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Monthly Update Report for Trials Started in December 2025


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1: Summary data from new trials identified for Leukemia and Lymphoma.


Overview

Number of Trials: 24

These 24 trials span diverse hematologic malignancies and supportive care interventions. The majority focus on leukemias (AML, ALL, CLL) and lymphomas (NHL, HL, T-cell lymphomas), with several exploring CAR-T therapy, novel targeted agents (menin inhibitors, IDH inhibitors, BCL-2 inhibitors), and combination regimens. Experimental treatments include bispecific antibodies, engineered T-cells, IL-18 variants, and AI-driven decision support tools. Many trials emphasize relapsed/refractory disease, maintenance therapy, and post-transplant consolidation.

Common Criteria Across Trials

Common Inclusion

  • Age ≥18 years
  • Pathologically confirmed diagnosis of hematologic malignancy
  • ECOG or Karnofsky performance status ≤2 or ≥50-60
  • Adequate organ function (renal, hepatic, cardiac)
  • Measurable disease by imaging or laboratory criteria
  • Ability to provide informed consent
  • Effective contraception for patients of childbearing potential

Common Exclusion

  • Active uncontrolled infection
  • Pregnancy or breastfeeding
  • HIV, hepatitis B, or hepatitis C infection (unless controlled)
  • Prior malignancy within 2-5 years (except non-melanoma skin cancer)
  • Significant cardiac dysfunction (NYHA Class III/IV, LVEF <40-50%)
  • CNS involvement by malignancy (unless controlled)
  • Concurrent investigational therapy
  • Severe organ dysfunction or comorbidities precluding safe participation

Outcomes Summary

Primary Outcomes

Secondary Outcomes


2: Extracted Trials with New Information


Trials with Special Criteria

Genomic / Biomarker Trials

Unique or Unusual Criteria

Trials with Emerging Treatments


3: Individual Trial Overviews


NCT07249528

Voice as a Digital Biomarker of Neurotoxicity in CAR T-Cell Therapy

Organization/Sponsor: Memorial Sloan Kettering Cancer Center


Example patient: A 52-year-old English-speaking patient with relapsed diffuse large B-cell lymphoma, ECOG status 1, scheduled for axicabtagene ciloleucel infusion, owns a smartphone and has no speech or neurological impairments.

Phase N/A

Interventions

  • Diagnostic: Audio Tasks
    Summary: Digital voice recording tasks performed twice daily via smartphone to monitor for neurotoxicity in patients receiving CAR T-cell therapy; used as a potential biomarker for early detection of neurological adverse events (source: Web Search).

Key Inclusion

  • Pathologically confirmed non-Hodgkin lymphoma or multiple myeloma
  • Scheduled to receive FDA-approved CAR-T product (Axi-cel, Liso-cel, Tisa-cel, Ide-cel, Cilta-cel, or Brexu-cel)
  • Age ≥ 18 years
  • ECOG Performance Status ≤ 2
  • Smartphone ownership
  • Sufficient English proficiency for structured voice tasks
  • Ability to perform twice daily voice recordings
  • Ability to undergo daily neurologic assessments

Key Exclusion

  • Pre-existing neurological conditions that significantly impair speech
  • Severe dysarthria or expressive aphasia
  • Neurodegenerative disorders
  • History of significant speech or voice disorders
  • Vocal cord pathology (paralysis, nodules, polyps, granulomas, malignancies)
  • Recent vocal cord surgery or radiation
  • Severe hearing impairment interfering with voice assessments
  • Language barriers preventing reliable task completion

NCT07285044

Cancer CARE (Connected Access and Remote Expertise) Beyond Walls - Pilot, Phase 2 Clinical Trial to Evaluate Administration of Cancer-Directed Therapy in the Patient's Homes Versus in Clinic in the Florida Panhandle and Surrounding Areas

Organization/Sponsor: Mayo Clinic


Example patient: A 62-year-old woman with metastatic breast cancer receiving trastuzumab and pertuzumab, ECOG PS 1, living independently in the Florida Panhandle with Wi-Fi access and planning at least 12 more weeks of treatment.

Phase 2

Interventions

  • Procedure: Questionnaire Administration
    Summary: Administration of questionnaires to assess patient outcomes and experiences during at-home cancer therapy delivery (NCI Thesaurus).
  • Procedure: Cancer Therapeutic Procedure
    Summary: Standard-of-care cancer interventions including immunotherapy, chemotherapy, and supportive agents administered at home versus clinic for various malignancies (NCI Thesaurus).

Key Inclusion

  • Histologically confirmed malignancy receiving eligible standard-of-care treatment regimen
  • Age >= 18 years
  • ECOG performance status 0-3
  • Adequate tolerability of current treatment without significant drug reactions
  • Resides in Florida Panhandle area serviced by at-home healthcare supplier
  • Wi-Fi connection available at residence
  • Plans to continue eligible treatment for >= 12 weeks
  • Social stability appropriate for at-home program

Key Exclusion

  • Co-morbid systemic illnesses making patient inappropriate for study entry
  • Receiving investigational agent for primary neoplasm treatment
  • Requires continuous 24/7 assistance without available caregiver support
  • Current inpatient hospitalization excluding Advanced Care at Home program

NCT07097363

A Pilot Study of Epcoritamab With Dose Adjusted Etoposide, Cyclophosphamide, Vincristine, Doxorubicin, Prednisone and Rituximab (EPOCH-R) for Upfront Treatment of Aggressive B-Cell Non-Hodgkin Lymphomas Genomic-based

Organization/Sponsor: University of Washington


Example patient: A 52-year-old with newly diagnosed high-grade B-cell lymphoma with MYC and BCL2 translocations, ECOG 1, measurable mediastinal mass 3.5 cm, adequate organ function, no prior lymphoma treatment.

Phase N/A

Interventions

  • Drug: Epcoritamab
    Summary: A CD20-directed CD3 T-cell engager bispecific antibody that bridges tumor B cells to T cells, redirecting immune activity against lymphoma cells for treating relapsed/refractory B-cell lymphomas (FDA label, NCI Thesaurus).
  • Drug: Rituximab
    Summary: A chimeric anti-CD20 monoclonal antibody that triggers cytotoxic immune response against CD20-positive B cells for treating B-cell malignancies and autoimmune conditions (FDA label, NCI Thesaurus).
  • Drug: Etoposide
    Summary: A topoisomerase II inhibitor that binds and prevents DNA ligation, causing DNA breaks and apoptosis in cancer cells, used for testicular tumors and small cell lung cancer (FDA label, NCI Thesaurus).
  • Drug: Cyclophosphamide
    Summary: An alkylating agent converted to active metabolites that bind DNA and inhibit replication, used for lymphomas, leukemias, breast/ovarian cancer, and nephrotic syndrome (FDA label, NCI Thesaurus).
  • Drug: Doxorubicin
    Summary: An anthracycline that intercalates DNA and inhibits topoisomerase II, causing DNA damage and cell death, used for breast cancer, leukemias, lymphomas, and solid tumors (FDA label, NCI Thesaurus).
  • Drug: Prednisone
    Summary: A synthetic glucocorticoid that activates nuclear receptors to suppress immune and inflammatory responses, used for inflammatory, allergic, autoimmune, and neoplastic conditions (FDA label, NCI Thesaurus).
  • Diagnostic Test: Positron Emission Tomography
    Summary: Imaging technique measuring gamma radiation from positron-electron collisions using radionuclide tracers to reveal metabolically active tissue in fine detail (NCI Thesaurus).
  • Diagnostic Test: Fludeoxyglucose F-18
    Summary: A positron-emitting radiopharmaceutical glucose analog that localizes to areas of increased glucose metabolism for PET imaging in oncology, cardiology, and neurology (FDA label, NCI Thesaurus).
  • Diagnostic Test: Computed Tomography
    Summary: X-ray imaging method using computer reconstruction to create cross-sectional scans of body structures (NCI Thesaurus).
  • Diagnostic Test: Multigated Acquisition Scan
    Summary: Diagnostic test using radioactive tracer and gamma camera to evaluate ventricular pumping function with cardiac cycle-gated image acquisition (NCI Thesaurus).
  • Diagnostic Test: Echocardiography Test
    Summary: Ultrasound procedure using high-frequency sound waves to create images of the heart (NCI Thesaurus).
  • Procedure: Bone Marrow Biopsy
    Summary: Removal of core tissue with bone spicules and marrow for diagnosing hematopoietic disorders and evaluating tumor spread (NCI Thesaurus).
  • Procedure: Bone Marrow Aspiration
    Summary: Aspiration of immature hematopoietic elements and blood from bone marrow for evaluating hematopoietic disorders, infections, and cytogenetics (NCI Thesaurus).
  • Procedure: Biospecimen Collection
    Summary: Gathering of biospecimens for testing, diagnostic, propagation, treatment, or research purposes (NCI Thesaurus).

Key Inclusion

  • Untreated aggressive large B-cell lymphoma with adverse features suitable for DA-EPOCH-R
  • Eligible histologies include high-grade B-cell lymphoma with MYC/BCL2/BCL6 translocations, DLBCL NOS, primary mediastinal B-cell lymphoma, Burkitt lymphoma
  • Age ≥18 years
  • Measurable disease (≥1.5 cm nodal or ≥1.0 cm extranodal)
  • ECOG performance status 0-2
  • LVEF ≥50% on MUGA or echocardiogram
  • ANC ≥1,000/μL, platelets ≥75,000/mcL, hemoglobin ≥8 g/dL (except marrow infiltration)
  • Creatinine clearance ≥45 mL/min, bilirubin ≤1.5×ULN, AST/ALT ≤2.5×ULN

Key Exclusion

  • Contraindication to EPOCH-R components or anthracycline cumulative dose >400 mg/m²
  • Prior systemic treatment for lymphoma (except 1 cycle chemoimmunotherapy or limited steroids)
  • Prior organ transplantation
  • Grade >1 peripheral neuropathy or demyelinating Charcot-Marie-Tooth disease
  • Active bacterial, viral, fungal, or parasitic infection requiring systemic treatment
  • Positive HBsAg or detectable HBV DNA; positive HCV RNA
  • Known active CNS lymphoma
  • Significant cardiovascular disease (NYHA Class III/IV, MI within 6 months, unstable arrhythmias/angina)
  • Pregnancy or lactation

NCT07270978

Phase Ib Study of Anti-CD3 x Anti-CD33 Bispecific Antibody (CD33Bi) Armed Fresh Peripheral Blood Mononuclear Cells (CD33 FPBMC) in Patients With Measurable Residual Disease (MRD)+ Acute Myeloid Leukemia or Myelodysplastic Syndrome Genomic-based

Organization/Sponsor: University of Virginia


Example patient: A 62-year-old man with relapsed AML after 3 cycles of azacitidine/venetoclax, now with 8% bone marrow blasts that are CD33-positive by flow cytometry and persistent NPM1 mutation, ECOG 1, adequate organ function, not currently planned for transplant.

Phase 1

Interventions

  • Biological: CD33 FPBMC
    Summary: CD33 FPBMC consists of fresh peripheral blood mononuclear cells armed with anti-CD3 x anti-CD33 bispecific antibody, designed to redirect T cells to attack CD33-expressing myeloid leukemia cells in AML and MDS patients with measurable residual disease. Source: Web Search Summary.

Key Inclusion

  • Adults ≥18 years of age
  • AML with persistent/recurrent MRD (blasts ≥5% and <20%, or MFC ≥0.1%, or persistent mutations/cytogenetics)
  • Residual blasts must be CD33 positive
  • R/R MDS or MDS/MPN overlap syndromes with disease progression criteria
  • Prior treatment with at least 2 cycles azacitidine and venetoclax (AML) or 4 cycles hypomethylating agent (MDS)
  • LVEF ≥45%
  • ECOG performance status ≤2
  • For targetable mutations (IDH1, IDH2, KMT2A, FLT3): receipt of or decision not to receive associated inhibitor

Key Exclusion

  • Prior treatment with anti-CD33 therapy
  • Actively being considered for stem cell transplant
  • AST or ALT >3x ULN, total bilirubin >1.5x ULN
  • Absolute lymphocyte count <300/microliter
  • Creatinine clearance <30 mL/min
  • Platelet refractory with platelets <30,000/microliter
  • Active serious infection not controlled by antibiotics
  • Known HIV with detectable viral load or active hepatitis B/C

NCT07228273

A Phase II Randomized Clinical Trial of Venetoclax Combined With FLAG IDA Induction and Consolidation Compared to Standard of Care for Newly Diagnosed Patients With Acute Myeloid Leukemia Genomic-based

Organization/Sponsor: OHSU Knight Cancer Institute


Example patient: A 52-year-old with newly diagnosed AML, ECOG status 1, adequate organ function, no FLT3 ITD mutation, willing to proceed to stem cell transplant after induction chemotherapy.

Phase II

Interventions

  • Drug: Venetoclax
    Summary: Selective BCL-2 inhibitor that induces apoptosis in leukemic cells by binding BCL-2 proteins and restoring apoptotic processes; FDA-approved for CLL/SLL and newly diagnosed AML in combination with hypomethylating agents (FDA label, NCI Thesaurus).
  • Drug: Idarubicin
    Summary: Anthracycline topoisomerase inhibitor that intercalates DNA and inhibits topoisomerase II, preventing DNA replication and RNA transcription; indicated for AML in combination therapy with high lipophilicity for enhanced cell penetration (FDA label, NCI Thesaurus).
  • Drug: Fludarabine
    Summary: Fluorinated nucleoside analog that inhibits DNA polymerase, ribonucleotide reductase, and DNA primase through its active triphosphate metabolite, interrupting DNA synthesis; FDA-approved for refractory B-cell CLL (FDA label, NCI Thesaurus).
  • Drug: Cytarabine
    Summary: Antimetabolite nucleoside analog that competes with cytidine for DNA incorporation and inhibits DNA polymerase, halting DNA replication in S phase; indicated for acute leukemias in combination regimens (FDA label, NCI Thesaurus).
  • Drug: Daunorubicin
    Summary: Anthracycline antibiotic that exhibits cytotoxic activity through topoisomerase-mediated DNA interaction, inhibiting DNA replication, repair, and protein synthesis; indicated for remission induction in acute leukemias (FDA label, NCI Thesaurus).
  • Procedure: Questionnaire Administration
    Summary: The act of having an individual fill out a questionnaire for data collection purposes (NCI Thesaurus).
  • Procedure: Multigated Acquisition Scan
    Summary: Diagnostic nuclear imaging test using radioactive tracer and gamma camera to evaluate ventricular pumping function with gated image acquisition at different cardiac cycle points (NCI Thesaurus).
  • Procedure: Echocardiography Test
    Summary: Ultrasound imaging procedure using high-frequency sound waves to create images of the heart for functional assessment (NCI Thesaurus).
  • Procedure: Bone Marrow Biopsy
    Summary: Removal of core tissue containing bone spicules and hematopoietic elements from hip area for diagnosis and evaluation of hematopoietic disorders including leukemias and lymphomas (NCI Thesaurus).
  • Procedure: Bone Marrow Aspiration
    Summary: Aspiration of immature hematopoietic elements and blood from bone marrow for evaluation of hematopoietic disorders, infectious diseases, and cytogenetic studies (NCI Thesaurus).
  • Procedure: Biospecimen Collection
    Summary: The act or process of gathering biospecimens for testing, diagnostic, propagation, treatment, or research purposes (NCI Thesaurus).

Key Inclusion

  • Age 18 to ≤65 years
  • Newly diagnosed, previously untreated AML or MDS with marrow blasts ≥10% per ELN22 criteria
  • ECOG performance status 0-1
  • Willingness to undergo hematopoietic stem cell transplant
  • Adequate organ function including creatinine clearance >40 mL/min
  • AST/ALT ≤3x ULN, total bilirubin ≤1.5x ULN unless due to leukemic involvement
  • Ability to take oral medications or via feeding tube
  • Negative pregnancy test and use of contraception for persons of childbearing potential

Key Exclusion

  • t(15;17) acute promyelocytic leukemia or FLT3 ITD or core binding factor mutations
  • Active CNS involvement with AML
  • WBC >25 x 10⁹/L
  • NYHA class III/IV heart failure or LVEF <40%
  • Prior BCL-2 inhibitor treatment within 12 months
  • Active malignancy within previous 5 years
  • Uncontrolled infection or active hepatitis B/C with detectable viral load
  • Use of strong/moderate CYP3A4 or P-gp inhibitors/inducers within specified timeframes

NCT07226934

Feasibility and Preliminary Efficacy of GPT-QPL: An AI-Generated, Personalized Question Prompt List Intervention for Patients With Hematologic Cancers

Organization/Sponsor: Washington University School of Medicine


Example patient: A 52-year-old patient with Non-Hodgkin Lymphoma receiving R-CHOP chemotherapy every three weeks at the outpatient lymphoma clinic, able to read English and use email independently.

Phase N/A

Interventions

  • Behavioral: GPT-QPL
    Summary: AI-generated personalized question prompt list that tailors questions based on patient profiles to enhance patient-provider communication and support for hematologic cancer patients. Source: Web Search.

Key Inclusion

  • Documented diagnosis of lymphoma (ICD-10 C81-C88) or multiple myeloma (ICD C90.0-C90.02)
  • Scheduled follow-up appointment at participating outpatient oncology clinic within next month
  • Undergoing chemotherapy for curative or disease management purposes every 1-4 weeks
  • Multiple myeloma patients must be in induction phase of treatment
  • Age 20-99 at enrollment
  • Able to consent to medical care independently
  • No visual impairments or English literacy limitations
  • Able to use email for electronic consent

Key Exclusion

  • Age 18-19 or older than 99
  • Has legally authorized representative
  • Documented diagnosis of cognitive impairment
  • Chemotherapy regimens delivered solely for palliative purposes
  • Visual impairments interfering with reading electronic or paper materials
  • English literacy limitations
  • Unable to use email

NCT07220993

Novel Unedited Allo Cell Therapy For High Risk T-Cell Malignancies Using CD7-Specific Car Expressed On T Cells (NEO-CRIMSON)

Organization/Sponsor: Baylor College of Medicine


Example patient: A 52-year-old with relapsed T-cell acute lymphoblastic leukemia 90 days post-allogeneic HSCT from a matched related donor, with 65% CD7-positive blasts, Karnofsky score 70%, no active GVHD, and suitable for second transplant.

Phase N/A

Interventions

  • Biological: CD7.CAR/28zeta T Cells
    Summary: Allogeneic T-lymphocytes genetically engineered to express a chimeric antigen receptor targeting CD7 antigen, linked to CD28 and CD3-zeta co-stimulatory domains, enabling specific recognition and lysis of CD7-expressing tumor cells in T-cell malignancies (NCI Thesaurus).

Key Inclusion

  • Recurrent T-ALL, T-LL, or T-NHL relapsed post-allogeneic related donor HSCT
  • CD7-positive tumor (≥20% CD7+ blasts by flow cytometry or immunohistochemistry)
  • Age ≤75 years old
  • Suitable for allogeneic HSCT with suitable donor identified
  • Karnofsky or Lansky score ≥60%
  • ≥60 days post-allogeneic HSCT at time of treatment
  • Available partially-HLA matched allogeneic EBV-specific T cell line
  • Prior allogeneic donor available for CD7.CAR T-cell manufacture

Key Exclusion

  • Active GVHD >Grade II or chronic GVHD >mild global severity
  • Corticosteroids >0.5mg/kg prednisone equivalent
  • Immunosuppressive treatment for GVHD within 28 days
  • Clinically significant infection or uncontrolled viral reactivation
  • CNS-3 disease or CNS abnormalities
  • LVEF <50% or cardiac dysfunction NYHA III/IV
  • Pregnant or lactating
  • Tumor location causing potential airway obstruction

NCT07225439

Phase I Clinical Trial of Rituximab (Rtx) and Tafasitamab in Combination With Allogeneic NK Cells for Treatment of Relapsed/Refractory (r/r) B-cell Non-Hodgkin Lymphoma

Organization/Sponsor: Case Comprehensive Cancer Center


Example patient: A 62-year-old with relapsed diffuse large B-cell lymphoma after three prior therapies including rituximab and CAR-T, with CD20-positive disease on recent biopsy, ECOG 1, and adequate organ function.

Phase 1

Interventions

  • Chemotherapy: Fludarabine/cyclophosphamide
    Summary: Lymphodepleting chemotherapy that induces apoptosis through DNA damage and immune modulation, used for treating chronic lymphocytic leukemia and indolent lymphomas (Web Search).
  • Biological: Interleukin-2
    Summary: Recombinant cytokine that binds IL-2 receptors to activate T cells and trigger gene expression, inducing T cell-mediated tumor regression (NCI Thesaurus).
  • Biological: Tafasitamab
    Summary: Humanized anti-CD19 monoclonal antibody with Fc-engineered domain that enhances ADCC and ADCP to deplete CD19-expressing B-cells in relapsed/refractory DLBCL and follicular lymphoma (FDA label, NCI Thesaurus).
  • Biological: Rituximab
    Summary: Chimeric anti-CD20 monoclonal antibody that triggers cytotoxic immune response against CD20-positive B-cells, indicated for B-cell malignancies including NHL and CLL (FDA label, NCI Thesaurus).
  • Biological: Allogeneic NK cells
    Summary: Natural killer cells from donor that kill cancer cells without specific antigen recognition by overcoming MHC class I inhibition, showing 92.9% response in lymphoma trials (Web Search).

Key Inclusion

  • Age 18 years or older
  • B-cell NHL including DLBCL, HGBCL, PMBCL, FL, CLL/SLL, MZL, or MCL
  • Measurable disease by Lugano 2014 or iwCLL 2018 criteria
  • Relapsed/refractory after two or more lines of systemic therapy
  • Prior CD19 and/or CD20 directed therapies
  • CD19 and/or CD20 expression on post-treatment relapse biopsy
  • ECOG Performance Status ≤2
  • Adequate organ function including cardiac ejection fraction ≥45%

Key Exclusion

  • Second active malignancy that would confound toxicity assessment
  • NYHA class III-IV congestive heart failure
  • Myocardial infarction or stroke within 6 months
  • Active hepatitis B or C infection (PCR positive)
  • Active CNS or leptomeningeal lymphoma involvement
  • History of CNS involvement without 90-day documented remission
  • Active autoimmune disease requiring systemic immunosuppression
  • Myelodysplastic syndrome features on bone marrow biopsy

NCT07283822

A Phase II Trial of JAK Inhibitor Added to Immunotherapy for Treatment of Relapsed/Refractory T-cell Lymphoma and 9p Amplified Lymphomas Genomic-based

Organization/Sponsor: Columbia University


Example patient: A 52-year-old with relapsed peripheral T-cell lymphoma-NOS after two prior systemic therapies including brentuximab vedotin, ECOG 1, adequate organ function, and no autoimmune disease.

Phase 2

Interventions

  • Biological: Pembrolizumab
    Summary: Humanized monoclonal IgG4 antibody blocking PD-1 receptor, enhancing T-cell-mediated immune responses against tumors; FDA-approved for multiple solid and hematologic malignancies including lymphomas (FDA label, NCI Thesaurus).
  • Drug: Ruxolitinib
    Summary: Oral JAK1/JAK2 inhibitor reducing inflammation and cellular proliferation via JAK-STAT pathway inhibition; FDA-approved for myelofibrosis, polycythemia vera, and graft-versus-host disease (FDA label, NCI Thesaurus).

Key Inclusion

  • Histologically confirmed relapsed/refractory HL, PMBCL, GZL, or TCL (PTCL, CTCL subtypes)
  • At least one prior systemic therapy; CD30+ ALCL/PTCL must have received CD30-directed therapy
  • Age ≥18 years
  • Measurable disease with PET-CT within 4 weeks
  • ECOG performance status 0-2
  • ANC ≥1000/μL, platelets ≥70,000/μL (≥50,000 if bone marrow involvement), hemoglobin ≥8 g/dL
  • Creatinine clearance ≥30 ml/min, bilirubin ≤2x ULN, AST/ALT ≤2.5x ULN
  • No chemotherapy/immunotherapy within 2 weeks; recovered from adverse events to grade ≤1

Key Exclusion

  • Diagnosis of Adult T-Cell Leukemia/Lymphoma (ATLL)
  • Autoimmune disease requiring systemic treatment
  • Chronic systemic steroids >10mg daily prednisone equivalent
  • HL/PMBCL patients eligible and agreeable to autologous stem cell transplant; PMBCL eligible for CAR-T
  • Solid organ transplant requiring active immunosuppression
  • Severe hypersensitivity (≥Grade 3) to pembrolizumab or ruxolitinib
  • Pregnant, breastfeeding, or unwilling to use contraception
  • Major cardiac events (MI, stroke) within 6 months

NCT07053020

A Phase 1b/2 Open-label, Dose-ranging Safety and Efficacy Study of Oral Cladribine in Patients With Acute Myeloid Leukemia (AML) Genomic-based

Organization/Sponsor: M.D. Anderson Cancer Center


Example patient: A 65-year-old man with newly diagnosed AML with monocytic phenotype (AML-M5), adequate organ function including LVEF 55%, no active infections, and willing to use contraception during treatment.

Phase 1, Phase 2

Interventions

  • Drug: Oral Cladribine
    Summary: A chlorinated purine nucleoside analog and antimetabolite that incorporates into DNA causing strand breaks, depleting NAD and ATP, and inducing apoptosis; selectively toxic to lymphocytes and monocytes due to resistance to adenosine deaminase (FDA label, NCI Thesaurus).
  • Drug: Cytarabine
    Summary: An antimetabolite cytidine analog with modified arabinose sugar that inhibits DNA polymerase and competes with cytidine for DNA incorporation, halting replication during S phase; used in acute leukemia combination regimens (FDA label, NCI Thesaurus).

Key Inclusion

  • Relapsed and/or refractory AML (Part 1 dose-escalation)
  • Newly diagnosed therapy-related AML from prior MDS treated with hypomethylating agents (Cohort A)
  • Newly diagnosed AML aged ≥60 years with monocytic phenotype (AML-M5) or RAS mutations (Cohort B)
  • Adequate renal function (serum creatinine ≤2x ULN)
  • Adequate hepatic function (bilirubin ≤2x ULN, AST/ALT ≤2.5x ULN or ≤5x ULN if leukemic involvement)
  • Left ventricular ejection fraction ≥50%
  • Effective contraception during treatment and 90 days after last dose

Key Exclusion

  • Uncontrolled infection or symptomatic congestive heart failure
  • Unstable angina or cardiac arrhythmia
  • History of allergic reactions to study compounds
  • Inability to swallow oral medications without altering formulation
  • Psychiatric illness or social situations limiting compliance
  • Pregnancy or breastfeeding

NCT05416554

Long-term Cognitive, Neuropsychiatric and Functional Outcomes in Adults Who Have Received Chimeric Antigen-Receptor T-Cell (CAR-T) Therapy for Aggressive Lymphoma at Stanford - A Pilot Study

Organization/Sponsor: Stanford University


Example patient: A 52-year-old English-speaking adult with aggressive lymphoma who received Axicabtagene ciloleucel CAR-T therapy 8 months ago at Stanford, is not on chemotherapy, has no progressive disease, and is able to participate in cognitive testing.

Phase N/A

Interventions

  • Procedure: Neuropsychological testing
    Summary: Performance-based assessment of cognitive functioning used to examine cognitive consequences of brain damage, brain disease, and severe mental illness (NCI Thesaurus).

Key Inclusion

  • 18 years or older
  • Treated with Axicabtagene ciloleucel CAR-T therapy at Stanford or elsewhere with Stanford follow-up
  • At least 6 months from CAR-T infusion
  • Fluent in English
  • Able to attend in-person or remote video testing

Key Exclusion

  • Concurrent enrollment in CAR-T therapeutics research study
  • Unable to participate in testing due to severe cognitive or physical limitation
  • Actively receiving chemotherapy
  • Progressive cancer

NCT05974202

Augmenting Cognitive-behavioral Therapy With rTMS of the Medial Prefrontal and Anterior Cingulate Cortices for the Treatment of Cocaine Use Disorder

Organization/Sponsor: New York State Psychiatric Institute


Example patient: A 34-year-old treatment-seeking man with moderate cocaine use disorder using cocaine 12 days in the past month, no other substance dependence, no psychiatric comorbidities, and no seizure history.

Phase N/A

Interventions

  • Device: Sham H7-coil repetitive transcranial magnetic stimulation (rTMS)
    Summary: Placebo control device mimicking active brain stimulation without therapeutic effect; used as comparator in trials studying depression, psychosis, and anxiety disorders (NCI Thesaurus).
  • Device: Active H7-coil repetitive transcranial magnetic stimulation (rTMS)
    Summary: Non-invasive brain stimulation therapy targeting medial prefrontal and anterior cingulate cortices; studied for depression, psychosis, anxiety, and substance use disorders (NCI Thesaurus).

Key Inclusion

  • Age 22-65 years
  • Current moderate/severe cocaine use disorder (DSM-5)
  • Treatment-seeking for cocaine use
  • Used cocaine at least 9 days in past 28 days with weekly use
  • Agree to moderate alcohol consumption limits
  • Women must use contraception

Key Exclusion

  • Current moderate/severe major depressive episode, bipolar disorder, schizophrenia, or psychotic disorder
  • Hamilton Depression Rating Scale >17 or Young Mania Rating Scale >10
  • Current moderate/severe other substance use disorder (except tobacco)
  • History of seizures including drug/alcohol withdrawal seizures
  • Medications lowering seizure threshold (bupropion, antipsychotics, lithium, tricyclics)
  • Implanted devices (pacemakers, stimulators, cochlear implants)
  • Metal implants or claustrophobia prohibiting MRI scanning
  • Significant suicide risk or suicidal behavior in past 3 months

NCT07216443

A Phase 2 Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Organization/Sponsor: Orca Biosystems, Inc.


Example patient: A 62-year-old with AML in first complete remission, Karnofsky score 70%, with an 8/8 HLA-matched sibling donor, deemed unsuitable for myeloablative conditioning due to cardiac comorbidities, planned for reduced intensity conditioning alloHCT.

Phase 2

Interventions

  • Biological: Orca-T
    Summary: A partially engineered T-regulatory cell donor graft depleted of T-cells then enriched with conventional T-cells, Tregs, and CD34+ stem cells from matched donors to induce tolerance in allogeneic hematopoietic stem cell transplantation for hematologic malignancies. Source: NCI Thesaurus.

Key Inclusion

  • Age ≥18 years
  • AML or mixed phenotype leukemia in CR or CRi, or MDS with ≤10% bone marrow blasts
  • Planned reduced intensity or nonmyeloablative conditioning regimen
  • 8/8 HLA-matched related or unrelated donor
  • eGFR ≥30 mL/min, cardiac ejection fraction ≥40%, DLCO ≥40%
  • Deemed ineligible for fully myeloablative alloHCT
  • Karnofsky performance score ≥60%
  • Negative pregnancy test in persons of childbearing potential

Key Exclusion

  • Prior allogeneic hematopoietic cell transplantation
  • Currently receiving immunosuppressive therapy (except ≤10 mg/day corticosteroids)
  • Planned T-cell depletion or donor lymphocyte infusion
  • Recipient-positive antidonor HLA antibodies against mismatched allele
  • HCT-CI ≥6 for RIC cohort
  • Uncontrolled infection or seropositive for HIV, HTLV, hepatitis B, or untreated HCV
  • Known allergy to tacrolimus or iron dextran
  • Pregnant, breastfeeding, or unwilling to use contraception for 1 year post-transplant

NCT07098364

A Phase 1/2 Open-Label Study Evaluating the Safety and Efficacy of ST-067 in Combination With CD19-Directed CAR T-Cell Therapy in Patients With Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL) Genomic-based

Organization/Sponsor: Fred Hutchinson Cancer Center


Example patient: A 52-year-old man with diffuse large B-cell lymphoma relapsed after three prior therapies including chemotherapy and immunotherapy, with CD19-positive disease on biopsy, Karnofsky score 70%, adequate cardiac and pulmonary function, and no prior CAR T-cell treatment.

Phase 1, Phase 2

Interventions

  • Procedure: Biospecimen Collection
    Summary: Collection of biological samples for testing, diagnostic, propagation, treatment or research purposes (NCI Thesaurus).
  • Diagnostic: X-Ray Imaging
    Summary: Radiographic imaging using x-ray emission to form images of internal structures (NCI Thesaurus).
  • Diagnostic: Positron Emission Tomography
    Summary: Imaging technique measuring gamma radiation from positron-electron collisions using radionuclide tracers like FDG to reveal metabolically active tissue (NCI Thesaurus).
  • Diagnostic: Multigated Acquisition Scan
    Summary: Diagnostic test evaluating ventricular pumping function using radioactive tracer and gated gamma camera imaging (NCI Thesaurus).
  • Procedure: Lymphodepletion Therapy
    Summary: Destruction of lymphocytes prior to immunotherapy to prepare the immune system for CAR T-cell infusion (NCI Thesaurus).
  • Procedure: Lumbar Puncture
    Summary: Invasive procedure using hollow needle through intervertebral space to sample cerebrospinal fluid or administer medication (NCI Thesaurus).
  • Biological: Lisocabtagene Maraleucel
    Summary: CD19-directed autologous CAR T-cell therapy with 4-1BB costimulation and EGFRt marker for relapsed/refractory B-cell lymphomas; FDA-approved (FDA label, NCI Thesaurus).
  • Procedure: Leukapheresis
    Summary: Procedure collecting white blood cells from peripheral blood while returning remaining components to donor (NCI Thesaurus).
  • Diagnostic: Echocardiography Test
    Summary: Ultrasound imaging procedure using high-frequency sound waves to create images of heart structure and function (NCI Thesaurus).
  • Diagnostic: Computed Tomography
    Summary: X-ray imaging method using computer reconstruction to create cross-sectional scans of internal body structures (NCI Thesaurus).
  • Procedure: Bone Marrow Biopsy
    Summary: Core tissue biopsy from hip area to diagnose hematopoietic disorders and evaluate tumor spread (NCI Thesaurus).
  • Procedure: Bone Marrow Aspiration
    Summary: Aspiration of immature hematopoietic elements from bone marrow for evaluation of hematopoietic disorders and infections (NCI Thesaurus).
  • Drug: Vevoctadekin
    Summary: Engineered IL-18 variant that preferentially binds IL-18 receptor over IL-18BP to enhance T-cell persistence and NK cell activity against tumors (NCI Thesaurus).

Key Inclusion

  • Age ≥18 years with relapsed/refractory large B-cell lymphoma
  • At least 2 prior lines of systemic therapy with FDA-approved indication for liso-cel
  • FDG-avid disease on PET or pathology evidence of active disease
  • CD19 expression on tumor specimen or high likelihood based on histology
  • Karnofsky performance status ≥60%
  • Adequate organ function: ANC ≥1000, platelets ≥50,000, creatinine clearance >30 mL/min
  • LVEF ≥40% and pulmonary function FEV1/FVC ≥60%, DLCO ≥40%
  • Effective contraception required for women and men

Key Exclusion

  • Prior CD19 CAR T-cell therapy
  • Active GVHD or systemic GVHD therapy within 30 days
  • Systemic corticosteroids >20 mg/day prednisone within 72 hours of infusion
  • Active hepatitis B, hepatitis C, or HIV infection
  • Active autoimmune or inflammatory disorders requiring immunosuppression
  • NYHA class III/IV heart failure or cardiac events within 6 months
  • Active CNS parenchymal involvement by malignancy
  • History of solid organ transplantation

NCT07049432

A Phase I Study of N-803 Maintenance Therapy Following CD19 Directed CAR T-cell Therapy in Patients With Relapsed or Refractory B-cell Non-Hodgkin Lymphomas (CARMEN-803 Trial)

Organization/Sponsor: University of Utah


Example patient: A 52-year-old with relapsed diffuse large B-cell lymphoma who achieved partial response 30 days after receiving axicabtagene ciloleucel, has resolved cytokine release syndrome, ECOG status 1, and adequate organ function.

Phase 1

Interventions

  • Biological: N803
    Summary: Nogapendekin alfa inbakicept is an IL-15 receptor agonist fusion protein that activates NK cells, CD8+ T-cells, and memory T-cells to enhance anti-tumor immune response. It binds IL-2/IL-15 receptors, increasing cytotoxic lymphocyte levels and IFN-gamma secretion for tumor cell killing (NCI Thesaurus).

Key Inclusion

  • Age ≥18 years with histologically confirmed B-cell NHL
  • Received commercially approved CD19-directed CAR T-cell therapy per FDA label
  • Achieved CR or PR per Lugano Criteria on Day +30 post-CAR-T
  • ECOG Performance Status ≤2
  • ANC ≥750 cells/mm3, platelets ≥50,000 cells/mm3, hemoglobin ≥7 g/dL
  • Creatinine clearance ≥30 mL/min by Cockcroft-Gault
  • CRS and ICANS grade 0 at enrollment, off steroids, ≥7 days from tocilizumab
  • Negative pregnancy test for females of childbearing potential

Key Exclusion

  • Prior therapy with N-803, IL-2, or IL-15 based therapy
  • Receiving other investigational agents
  • Autoimmune disease requiring active treatment (except hypothyroidism, diabetes type 1)
  • Stroke or intracranial hemorrhage within 6 months
  • Myocardial infarction within 3 months or unstable angina within 2 months
  • LVEF <40% in prior 12 months or NYHA Grade ≥3 heart failure
  • Known HIV infection or active hepatitis B or C
  • Ongoing CAR T-cell toxicity interfering with study treatment per investigator

NCT06771830

A Rapid Diagnostic of Risk in Hospitalized Pediatric Patients to Improve Outcomes Using Machine Learning

Organization/Sponsor: University of Wisconsin, Madison


Example patient: A 12-year-old hospitalized pediatric patient with acute lymphoblastic leukemia being monitored with eCART for early detection of clinical deterioration.

Phase N/A

Interventions

  • Decision Support Tool: Pediatric eCART
    Summary: Pediatric eCART is a machine learning-based decision-support tool using electronic health records to predict life-threatening events in hospitalized children, quantifying disease severity and identifying high-risk patients for early intervention (Source: Web Search).

Key Inclusion

  • Pediatric patients under 18 years of age
  • Eligible for pediatric eCART scoring
  • Inpatient locations
  • UW Health nurses who interact with eCART during patient care

Key Exclusion

  • Patients ineligible for pediatric eCART scoring
  • Neonates and birth encounters
  • UW Health nurses no longer employed at UW Health

NCT07052994

A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients Genomic-based

Organization/Sponsor: M.D. Anderson Cancer Center


Example patient: A 7-year-old with relapsed AML harboring KMT2A rearrangement, ECOG 1, normal cardiac function, WBC 18,000/μL, and no active infections.

Phase 1

Interventions

  • Drug: Daunorubicin
    Summary: Anthracycline topoisomerase inhibitor that inhibits DNA replication, repair, and RNA/protein synthesis, used for remission induction in acute leukemias in combination with other anticancer drugs (FDA label, NCI Thesaurus).
  • Drug: Cytarabine
    Summary: Antineoplastic nucleoside metabolic inhibitor that disrupts DNA replication by inhibiting nucleic acid synthesis, used for acute leukemias and meningeal leukemia prophylaxis/treatment (FDA label).
  • Drug: Revumenib
    Summary: Oral menin inhibitor that blocks menin-KMT2A protein interaction, reducing downstream gene expression and inhibiting proliferation of KMT2A-rearranged leukemic cells in relapsed/refractory acute leukemia (FDA label, NCI Thesaurus).

Key Inclusion

  • Age 6 months to 21 years
  • ECOG performance status <2
  • Phase 1a: Relapsed/refractory AML or MPAL with KMT2Ar, NPM1c, NUP98r, or UBTF-ITD
  • Phase 1b: Frontline AML or MPAL with KMT2Ar, NUP98r, or UBTF-ITD
  • WBC <25,000/μL at enrollment
  • Ejection fraction >50%
  • Adequate hepatic and renal function
  • At least 14 days from prior treatment or 5 half-lives, whichever is shorter

Key Exclusion

  • Uncontrolled medical condition or psychiatric illness
  • Severe gastrointestinal or metabolic condition interfering with oral medication absorption
  • Concurrent active malignancy under treatment
  • Active hepatitis B, C, or HIV infection
  • Pregnant or breast-feeding
  • Active uncontrolled infection
  • Myocardial infarction, unstable angina, CHF Class >II, or CVA within 6 months
  • QTc >450 msec (males) or >470 msec (females)

NCT07177079

High-dose Ascorbate (HDA) in Combination With Azacitidine and Venetoclax (Aza/Ven) in Newly Diagnosed Acute Myeloid Leukemia (AML) Genomic-based

Organization/Sponsor: University of Iowa


Example patient: A 78-year-old man with newly diagnosed AML without favorable cytogenetics, ECOG performance status 2, ejection fraction 45%, and no prior AML treatment.

Phase N/A

Interventions

  • Drug: Decitabine
    Summary: Decitabine is a nucleoside metabolic inhibitor that incorporates into DNA and inhibits DNA methyltransferase, causing hypomethylation and intra-S-phase arrest, indicated for myelodysplastic syndromes (FDA label, NCI Thesaurus).
  • Drug: High-dose ascorbate
    Summary: High-dose ascorbate targets redox imbalance, epigenetic reprogramming, and oxygen vulnerabilities in cancer cells, enhancing immune activity with in vitro anticancer effects in leukemia (Web Search Summary).
  • Drug: Venetoclax
    Summary: Venetoclax is a selective BCL-2 inhibitor that binds BCL-2 proteins to restore apoptosis in tumor cells, indicated for CLL, SLL, and newly diagnosed AML in combination with hypomethylating agents (FDA label, NCI Thesaurus).
  • Drug: Azacitidine
    Summary: Azacitidine is a pyrimidine nucleoside analogue that inhibits DNA methyltransferase causing hypomethylation to activate tumor suppressor genes, indicated for myelodysplastic syndrome subtypes (FDA label, NCI Thesaurus).

Key Inclusion

  • Adults aged ≥18 unfit for intensive chemotherapy
  • Age ≥75 or ECOG performance 2-3
  • Newly diagnosed non-APL acute myeloid leukemia
  • Severe cardiac, pulmonary, renal, or hepatic disorder
  • No prior chemotherapy or hypomethylating agent for AML
  • Adequate organ function with AST/ALT ≤3.0x ULN
  • History of antecedent MDS without prior chemotherapy allowed
  • Therapy-related AML without prior hypomethylating agent or venetoclax allowed

Key Exclusion

  • Prior therapy for AML except hydroxyurea or low-dose cytarabine
  • Known hypersensitivity to ascorbate, azacitidine, decitabine, or venetoclax
  • Favorable cytogenetics: t(8;21), inv(16), bZIP CEBPA, KMT2A rearrangement
  • NPM1, IDH1, IDH2, FLT3-ITD, or FLT3-TKD mutations
  • Kidney disease needing dialysis or history of oxalate nephropathy
  • Primary hemochromatosis or ferritin >1000 ng/mL
  • Diabetes on short-acting insulin requiring daily glucose monitoring
  • G6PD deficiency or on warfarin/strong CYP3A4 inducers/inhibitors

NCT07014917

Randomized Phase II Study of Intermittent Versus Continuous Venetoclax Therapy With Acalabrutinib in Previously Untreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) and Variants of This Genomic-based

Organization/Sponsor: University of Cincinnati


Example patient: A 62-year-old man with newly diagnosed CLL with del(11q) by FISH, leukocytosis, lymphadenopathy, ECOG 1, adequate organ function, and no prior CLL therapy.

Phase 2

Interventions

  • Drug: Acalabrutinib
    Summary: Acalabrutinib is an oral Bruton's tyrosine kinase (BTK) inhibitor that blocks B-cell receptor signaling, preventing activation and survival of malignant B-cells in CLL, SLL, and MCL (FDA label, NCI Thesaurus).
  • Drug: Venetoclax
    Summary: Venetoclax is an oral BCL-2 inhibitor that restores apoptosis in tumor cells by binding BCL-2 proteins, indicated for CLL, SLL, and AML in combination regimens (FDA label, NCI Thesaurus).

Key Inclusion

  • Previously untreated CLL or SLL requiring therapy by IWCLL criteria
  • Age ≥18 years
  • ECOG performance status 0-2
  • ANC ≥1,000/mcL or ≥500/mcL if due to CLL marrow involvement
  • Platelets ≥30,000/mcL or ≥10,000/mcL if due to CLL marrow involvement
  • Creatinine clearance ≥30 mL/min
  • Adequate liver function (bilirubin ≤1.5x ULN, AST/ALT ≤3x ULN)
  • Atypical immunophenotype allowed if cytogenetics/genomics support CLL/SLL

Key Exclusion

  • Active Richter's transformation
  • CNS lymphoma history or ongoing
  • Major surgery within 30 days
  • Myocardial infarction or Class 3-4 heart failure within 6 months
  • Stroke or intracranial hemorrhage within 6 months
  • Requires warfarin or strong CYP3A inhibitors/inducers
  • Active hepatitis B or C infection
  • Unable to receive Prevnar vaccination

NCT07257419

CD45RA-depleted CD19-CAR T Cell Consolidation After TCRαβ+ T Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma

Organization/Sponsor: St. Jude Children's Research Hospital


Example patient: A 14-year-old with high-risk CD19+ B-cell ALL in second complete remission with adequate cardiac and renal function, performance score of 60, and an available haploidentical parent donor.

Phase N/A

Interventions

  • Device: CliniMACS System
    Summary: A proprietary flow cytometry system using anti-CD34 antibodies to isolate CD34+ cells from apheresis specimens for cell processing (NCI Thesaurus).
  • Biological: Filgrastim
    Summary: Recombinant human granulocyte colony-stimulating factor that stimulates neutrophil production and release from bone marrow to prevent neutropenia and mobilize stem cells (FDA label, NCI Thesaurus).
  • Drug: Melphalan
    Summary: Bifunctional alkylating agent that damages DNA at N7 position of guanine, inducing DNA cross-linkages and inhibiting tumor cell growth, used in conditioning regimens (FDA label, NCI Thesaurus).
  • Drug: Mesna
    Summary: Cytoprotective sulfhydryl compound that detoxifies toxic metabolites of ifosfamide and cyclophosphamide in the kidney to prevent hemorrhagic cystitis (FDA label, NCI Thesaurus).
  • Drug: Thiotepa
    Summary: Organophosphorus alkylating agent converted to ethylenimine groups that crosslink DNA at guanine N7 position, inhibiting DNA replication and inducing apoptosis (FDA label, NCI Thesaurus).
  • Drug: Fludarabine
    Summary: Fluorinated nucleoside analog that inhibits DNA polymerase, ribonucleotide reductase, and DNA primase through its active triphosphate metabolite, interrupting DNA synthesis (FDA label, NCI Thesaurus).
  • Drug: Cyclophosphamide
    Summary: Alkylating agent converted in liver to active metabolites that bind DNA and inhibit replication, providing antineoplastic and immunosuppressive effects (FDA label, NCI Thesaurus).
  • Biological: Anti-Thymocyte Globulin (Rabbit)
    Summary: Polyclonal antibody from rabbits immunized with human thymocytes that depletes T-lymphocytes by binding surface antigens, providing immunosuppression to prevent graft-versus-host disease (FDA label, NCI Thesaurus).

Key Inclusion

  • Age ≤21 years
  • High risk CD19+ B-cell ALL in CR1, CR2, CR3 or subsequent remission
  • Allogeneic transplantation is standard of care
  • Left ventricular ejection fraction >40% or shortening fraction ≥25%
  • Creatinine clearance or GFR ≥50 ml/min/1.73m2
  • Karnofsky or Lansky performance score ≥50
  • At least single haplotype matched (≥4 of 8) family donor available
  • Prior CNS leukemia must be treated and in CNS CR

Key Exclusion

  • Has suitable HLA-identical sibling or 12/12 matched unrelated donor available
  • Any other active malignancy
  • Prior allogeneic HCT at any time
  • Autologous HCT within previous 6 months
  • Pregnant or breast feeding
  • Severe uncontrolled bacterial, fungal or viral infection

NCT07304011

A Phase 2 Study of Olutasidenib in Combination With Azacitidine Followed by Olutasidenib Maintenance After Venetoclax Plus a Hypomethylating Agent Regimen (HMA-VEN) for IDH1-Mutated Acute Myeloid Leukemia (AML) Genomic-based

Organization/Sponsor: University of California, Davis


Example patient: A 67-year-old man with newly diagnosed IDH1-R132 mutated AML who achieved complete remission after 3 cycles of azacitidine-venetoclax, ECOG status 1, with normal organ function and no prior malignancies.

Phase 2

Interventions

  • Drug: Olutasidenib
    Summary: Olutasidenib is an oral IDH1-R132 inhibitor that blocks the mutated IDH1 enzyme, preventing formation of oncometabolite 2-hydroxyglutarate and promoting cellular differentiation in IDH1-mutated AML. FDA-approved for relapsed/refractory AML with susceptible IDH1 mutations (FDA label, NCI Thesaurus).
  • Drug: Azacitidine
    Summary: Azacitidine is a pyrimidine nucleoside analogue that inhibits DNA methyltransferase, reactivating silenced tumor suppressor genes and disrupting RNA function in myelodysplastic syndromes and AML (FDA label, NCI Thesaurus).

Key Inclusion

  • Pathologically documented AML with WHO or ICC criteria (excluding acute promyelocytic leukemia with t(15;17))
  • Achieved CR/CRi response to first-line HMA-Ven per ELN recommendations
  • Documented IDH1-R132 mutations (≥0.01%) in bone marrow or blood at diagnosis or after HMA-Ven initiation
  • Receiving first-line HMA-Ven with ≤4 cycles at enrollment
  • ECOG performance status ≤2
  • Age ≥18 years
  • Creatinine clearance ≥40 mL/min
  • ALT and AST ≤3× ULN, bilirubin ≤2× ULN

Key Exclusion

  • Prior IDH1 inhibitor targeted therapy
  • Prior AML therapy except HMA-Ven
  • Active malignancy within 12 months (with specified exceptions)
  • Previous allogeneic HSCT <100 days, active GvHD, or immunosuppression >20 mg methylprednisolone equivalent
  • Baseline QTcF >480 msec (except bundle branch block)
  • Congestive heart failure (NYHA Class III/IV) or myocardial infarction within one year
  • Active uncontrolled infections requiring systemic therapy
  • Pregnancy, breastfeeding, or unwillingness to use contraception

NCT06707493

A Randomized, Placebo-Controlled Phase 2 Study of IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Acute Myeloid Leukemia Following Allogeneic Stem Cell Transplantation Genomic-based

Organization/Sponsor: Massachusetts General Hospital


Example patient: A 52-year-old woman with IDH1(R132)-mutant AML in remission, 60 days post-allogeneic stem cell transplant with 85% donor chimerism, ECOG 1, no active GVHD, and adequate organ function.

Phase 2

Interventions

  • Drug: Ivosidenib
    Summary: Ivosidenib is an oral IDH1 inhibitor that blocks mutated IDH1 enzyme, preventing production of oncometabolite 2-hydroxyglutarate and promoting cellular differentiation in IDH1-mutant AML. FDA-approved for IDH1-mutant AML, myelodysplastic syndromes, and cholangiocarcinoma; induces CYP2C9 and CYP3A4 enzymes. Sources: FDA label, NCI Thesaurus.
  • Drug: Placebo
    Summary: Inactive compound identical in appearance to ivosidenib, used as control to distinguish drug action from suggestive effects in this randomized trial. Source: NCI Thesaurus.

Key Inclusion

  • IDH1(R132)-mutant acute myeloid leukemia confirmed pathologically
  • Ages 18 to 75 years
  • Undergoing allogeneic hematopoietic stem cell transplantation
  • ECOG performance status ≤2
  • ANC ≥1000/µL without growth factor support
  • Platelet count ≥50,000/µL without transfusion support
  • LVEF ≥40% by MUGA or echocardiogram
  • Treatment start between day 45 and 90 post-transplant with ≥70% donor chimerism

Key Exclusion

  • Prior allogeneic hematopoietic stem cell transplants
  • Morphologically relapsed or refractory disease within 42 days of entry
  • Active hepatitis B or hepatitis C
  • Congestive heart failure NYHA class 3 or 4 or LVEF <40%
  • QTc interval ≥450 ms or history of ventricular/life-threatening arrhythmias
  • Acute GVHD requiring ≥0.5mg/kg/day prednisone equivalent within one week of treatment
  • Evidence of relapsed/recurrent/residual disease at treatment start
  • Systemic infection requiring IV antibiotics within 7 days of treatment start

NCT06446661

Improving Oral Chemotherapy Adherence in Maintenance for Adolescents and Adults With Acute Lymphoblastic Leukemia Using Text Messages

Organization/Sponsor: University of Chicago


Example patient: A 22-year-old with newly diagnosed ALL about to start maintenance therapy with mercaptopurine and methotrexate on a CALGB 10403-based regimen who owns a cell phone.

Phase N/A

Interventions

  • Behavioral: Low Intensity Text Messaging
    Summary: Regular SMS reminders to help patients manage chemotherapy side effects and improve adherence to oral maintenance therapy, targeting engagement and reducing time burden (Summary of Web Search).
  • Behavioral: No Text Messaging
    Summary: Control arm with no text message intervention for comparison of adherence to oral chemotherapy (Summary of Web Search).
  • Behavioral: High Intensity
    Summary: High-intensity text messaging intervention with more frequent reminders and support to enhance adherence and symptom management during maintenance therapy (Summary of Web Search).

Key Inclusion

  • Age 15-39 years at initial ALL diagnosis
  • Diagnosed with acute lymphoblastic leukemia
  • Receiving pediatric-based regimen with maintenance mercaptopurine and methotrexate
  • Enrollment prior to start of maintenance phase

Key Exclusion

  • Patient or caregiver lacks cell phone that receives text messages
  • Patient does not wish to participate