Sophic Logo gordian knotPediatric Cancer Clinical Trials Intelligence

Monthly Update Report - October 2025


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Important Notice:
Sophic does not practice medicine nor provide medical advice. The Sophic Starlight Cancer Clinical Trials Intelligence Report is intended solely as an educational resource that provides access to publicly available clinical trial data integrated within Sophic’s proprietary knowledgebase and summarized with AI. This report is not a substitute for professional medical advice, diagnosis, or treatment.

1: Summary data from new trials identified for Pediatric Cancer.


Overview

Number of Trials: 20

These 20 trials span diverse pediatric and young adult cancers, including leukemias, lymphomas, solid tumors (osteosarcoma, Ewing sarcoma, neuroblastoma, rhabdomyosarcoma), and lung cancers. Interventions range from novel CAR-T cell therapies, checkpoint inhibitors, targeted small molecules, and chemotherapy combinations to supportive care innovations like medically tailored meals, telegenetics, and digital mental health tools. Many trials focus on relapsed/refractory disease, precision medicine via genetic testing, and immunotherapy. Several emphasize adolescent and young adult (AYA) populations, addressing unique psychosocial and access challenges.

Common Criteria Across Trials

Common Inclusion

  • Age ranges from infants (≥1 month) to young adults (up to 39 years)
  • Histologically confirmed cancer diagnosis
  • Relapsed or refractory disease after standard therapy
  • Adequate organ function (renal, hepatic, cardiac, pulmonary)
  • Performance status (Karnofsky/Lansky ≥50-60, ECOG ≤2)
  • Measurable or evaluable disease
  • Recovery from prior therapy toxicities
  • Informed consent/assent

Common Exclusion

  • Pregnancy or breastfeeding
  • Active uncontrolled infection
  • HIV, hepatitis B/C infection
  • CNS disease (active or uncontrolled)
  • Prior allogeneic stem cell transplant (in some trials)
  • Severe cardiac dysfunction
  • Concurrent malignancy
  • Uncontrolled autoimmune disease
  • Use of systemic corticosteroids or immunosuppressants

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


NCT07091617

AYA Access Study: An Enhanced eHealth and Chat-Bot Enabled Delivery Model for Clinical Genetic Services in Community AYA Cancer Patients Genomic-based

Organization/Sponsor: Alliance for Clinical Trials in Oncology


Example patient: A 28-year-old English-speaking woman with metastatic breast cancer and family history of BRCA mutations, currently in treatment and cognitively intact, seeking genetic counseling.

Phase N/A

Interventions

  • Behavioral: Survey Administration
    Summary: Survey administration gathers patient-reported outcomes and experiences in clinical trials, assessing cognitive and behavioral impacts of treatment (NCI Thesaurus, Web Search).
  • Behavioral: Interview
    Summary: Interview method collects patient background, personal details, and opinions on specific subjects posed by the interviewer (NCI Thesaurus, Web Search).
  • Behavioral: Patient Navigation
    Summary: Patient navigation guides patients through healthcare systems, reducing barriers to timely screening, diagnosis, treatment, and supportive care, boosting clinical trial participation (NCI Thesaurus, Web Search).
  • Behavioral: Educational Intervention
    Summary: Educational activities intended to prevent disease or alter disease course by improving understanding and participation through enhanced communication and support (NCI Thesaurus, Web Search).
  • Behavioral: Internet-Based Intervention
    Summary: Online programs using internet platforms to alter behavior, targeting mental health and physical activity to improve wellbeing in cancer patients (NCI Thesaurus, Web Search).
  • Diagnostic: Genetic Testing
    Summary: DNA testing detects genetic alterations or defects predisposing to disease, identifying tumor-specific mutations to tailor precision medicine treatments (NCI Thesaurus, Web Search).
  • Other: Telemedicine
    Summary: Telecommunications technology provides remote health care services, enhancing monitoring, reducing travel, and improving trial access and efficiency (NCI Thesaurus, Web Search).

Key Inclusion

  • Age 18-39 years at enrollment
  • AYA cancer patients and survivors at any stage
  • Cancer diagnosis at any age ≤39 years
  • Able to speak and read English or Spanish
  • Meet NCCN guidelines for genetic testing assessment

Key Exclusion

  • Known diagnosis of dementia or cognitive impairment
  • Impaired decision-making capacity
  • Psychiatric or developmental disorder affecting cognitive or emotional functions

NCT07148895

A Prospective, Randomized, Multi-Center Trial Assessing Post-Operative Outcomes Following Pediatric Sistrunk Procedures With or Without Drain Placement

Organization/Sponsor: University of Rochester


Example patient: A 7-year-old child with a 2cm midline neck thyroglossal duct cyst scheduled for Sistrunk procedure with no bleeding disorders or active infection.

Phase N/A

Interventions

  • Procedure: No post-surgical drain
    Summary: Standard practice avoiding drain placement to minimize infection risk and reduce complications after pediatric surgery, enhancing recovery by preventing fluid accumulation issues (Summary of Web Search).
  • Device: Post-surgical drain
    Summary: Hollow tube inserted at surgical site to remove excess fluid and prevent complications; drains fluid into collection device to manage accumulation after surgery (NCI Thesaurus, Summary of Web Search).

Key Inclusion

  • Children aged 18 and younger
  • Diagnosis of <3cm midline neck mass, tract or sinus
  • Patient scheduled for Sistrunk procedure
  • Patient admitted for overnight observation
  • Thyroglossal duct cyst confirmed on final pathology

Key Exclusion

  • Confirmed bleeding or immunodeficiency disorders
  • Lesions greater than 3cm on preoperative imaging
  • Lingually positioned lesions
  • Evidence of overt infection at time of surgery
  • Coexistent lesion excision
  • Entry into oropharynx noted during procedure
  • Revision surgery if prior formal Sistrunk performed

NCT07070219

A Single-Arm, Open-Label, Multi-Center, Phase 1b/ 2 Study to Evaluate the Safety, Efficacy, and Cellular Pharmacokinetic Profile of CTD402 in Participants With Relapsed/Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL) and Lymphoblastic Lymphoma (T-LBL) (TENACITY-01)

Organization/Sponsor: BIOHENG THERAPEUTICS US LLC


Example patient: A 14-year-old with relapsed T-ALL after three prior therapies, bone marrow blasts at 15%, Lansky score 70, with an available haploidentical donor and no active infections.

Phase 1b, Phase 2

Interventions

  • Biological: CTD402 CAR T Cell Injection
    Summary: Allogeneic, off-the-shelf, universal CAR-T cells targeting CD7 with CD7 knockout and HLA class II disruption to prevent fratricide and immune rejection. Expresses anti-CD7 CAR with 4-1BB and CD3 zeta domains for treating CD7-positive T-cell malignancies. Source: NCI Thesaurus.

Key Inclusion

  • Age ≥12 years
  • Body weight ≥40 kg
  • Relapsed/refractory T-ALL/LBL after ≥2 lines of therapy or first relapse within 12 months
  • Bone marrow lymphoblasts ≥5% or extramedullary disease
  • Eligible HLA-matched, haploidentical, or syngeneic donor available
  • Adequate organ function
  • Karnofsky PS ≥60 (age ≥16) or Lansky PS ≥60 (age <16)
  • Relapse ≥100 days post-allogeneic HSCT if applicable

Key Exclusion

  • Genetic syndromes with bone marrow failure
  • Active CNS involvement
  • NYHA class III/IV heart failure or recent cardiac events within 12 months
  • Primary immune deficiency
  • Uncontrolled infections
  • HIV, hepatitis C, or syphilis infection
  • Active or latent hepatitis B
  • EBV, CMV DNA or IgM positive at screening

NCT07197554

A Phase 1/1B Study of ST-01156, a Small Molecule RBM39 Degrader, in Patients With Advanced Solid Malignancies

Organization/Sponsor: SEED Therapeutics, Inc.


Example patient: A 19-year-old with metastatic Ewing sarcoma, ECOG status 1, with measurable lung lesions, adequate organ function, no prior RBM39 inhibitors, and no active brain metastases.

Phase 1

Interventions

  • Drug: ST-01156
    Summary: ST-01156 is a small molecule RBM39 degrader that targets the RBM39 splicing factor protein for degradation, modulating cellular RNA splicing responses in advanced solid tumors (Source: Summary of Web Search).

Key Inclusion

  • Age ≥18 years, or ≥16 years for Ewing Sarcoma or malignancies with biological rationale
  • Metastatic or locally advanced and unresectable solid tumor
  • At least 1 measurable lesion or evaluable disease per RECIST v1.1
  • ECOG performance status ≤2
  • Adequate organ function

Key Exclusion

  • Prior radiotherapy within 2 weeks of treatment
  • Known active CNS metastases or carcinomatous meningitis
  • Anticancer therapy or investigational agent within 14 days or 5 half-lives
  • Major surgery within 28 days
  • Unresolved toxicities from previous therapies except alopecia and peripheral neuropathy
  • Previously received a RBM39 inhibitor or degrader

NCT06654466

Enhancing Information Management for Young Adults After Genetic Cancer Risk Testing Genomic-based

Organization/Sponsor: Nest Genomics


Example patient: A 32-year-old English-speaking woman receiving care at Dana Farber Cancer Institute with a BRCA1 pathogenic variant identified on prior genetic testing, currently cancer-free and not undergoing active treatment.

Phase N/A

Interventions

  • Software Platform: Nest, an electronic medical record (EMR)-integrated software platform to deliver longitudinal, genetics-based care at scale.
    Summary: Nest is an AI-enabled platform integrating EMRs for precision medicine, using genomic data to inform clinical decisions and improve patient outcomes in genetics-based care (Source: Web Search).

Key Inclusion

  • Ages 18-49 years, inclusive
  • Previous cancer genetic testing with pathogenic or likely pathogenic variant
  • Increased risk of cancer warranting clinical management
  • English-speaking and -reading
  • Receiving care at Dana Farber Cancer Institute
  • Not in active cancer therapy at time of approach

Key Exclusion

  • Age <18 or >49 years
  • No genetic testing for hereditary cancer syndromes
  • Tested but no pathogenic or likely pathogenic variant identified
  • Non-English speaking and reading
  • Not receiving care at Dana Farber Cancer Institute
  • Active cancer with therapy in progress

NCT07144254

Study of Tegavivint, a TBL1 Inhibitor, With Gemcitabine in Patients With Relapsed or Refractory Osteosarcoma

Organization/Sponsor: Emory University


Example patient: A 14-year-old with relapsed osteosarcoma and pulmonary metastases after treatment with methotrexate, doxorubicin, cisplatin, and ifosfamide, with Karnofsky score 70, adequate organ function, and no CNS involvement.

Phase N/A

Interventions

  • Drug: Gemcitabine
    Summary: Gemcitabine is a nucleoside metabolic inhibitor and deoxycytidine analogue that disrupts DNA synthesis by incorporating into DNA and inhibiting ribonucleotide reductase, halting cancer cell growth. FDA-approved for ovarian, breast, lung, and pancreatic cancers; studied in pediatric refractory solid tumors. Sources: FDA label, NCI Thesaurus, Web Search.
  • Drug: Tegavivint
    Summary: Tegavivint is a small molecule inhibitor targeting TBL1 that disrupts Wnt/beta-catenin signaling by promoting beta-catenin degradation, reducing TCF4 activity and expression of cyclin D1, c-Myc, and survivin. Investigated for pediatric cancers with aberrant Wnt pathway activation. Sources: NCI Thesaurus, Web Search.

Key Inclusion

  • Histologically verified osteosarcoma at original diagnosis or relapse
  • Relapsed or refractory osteosarcoma after front-line treatment with at least 3 agents: methotrexate, doxorubicin, cisplatin, ifosfamide
  • Measurable or evaluable disease per RECIST (dose escalation requires evidence of disease)
  • Lansky or Karnofsky score ≥60, or ECOG ≤2
  • ANC ≥750/mm3, platelets ≥75,000/mm3
  • Creatinine clearance or GFR ≥70 ml/min/1.73 m2
  • Bilirubin ≤1.5x ULN, ALT ≤5x ULN
  • QTc ≤470 ms, no dyspnea at rest, pulse oximetry >93% on room air

Key Exclusion

  • Active intraparenchymal CNS osteosarcoma unless stable for ≥3 months
  • Pregnancy, breastfeeding, or refusal of two effective contraceptive methods
  • Concurrent investigational drugs or anti-cancer agents
  • Strong CYP3A4/5 inducers or inhibitors within 14 days
  • Bisphosphonates within 4 weeks or denosumab within 180 days
  • Prior solid organ or allogeneic stem cell transplantation
  • Metabolic bone disease or disorder affecting bone metabolism
  • Grade ≥2 hypocalcemia uncorrected by oral supplementation or vitamin D <20 ng/mL without supplementation

NCT05779930

Safety and Feasibility of On-Site Manufacture of CD19 CAR T Cells Using the CliniMACS Prodigy in Pediatric and Young Adult Patients With Relapsed/Refractory CD19 Positive Acute Lymphoblastic Leukemia and Non-Hodgkin's Lymphoma

Organization/Sponsor: Nationwide Children's Hospital


Example patient: A 12-year-old with B-cell ALL in third relapse after allogeneic stem cell transplant, CD19-positive disease confirmed by flow cytometry, Lansky score 60, adequate organ function, and no active infections.

Phase N/A

Interventions

  • Biological: CD19 specific Chimeric Antigen Receptor T Cell
    Summary: Autologous or allogeneic T-lymphocytes genetically modified to express a chimeric antigen receptor targeting CD19-positive cancers, particularly pediatric B-ALL and NHL. The therapy reprograms patient T cells to attack CD19-expressing cancer cells, showing high response rates with potential severe side effects. Sources: NCI Thesaurus, Web Search Summary.

Key Inclusion

  • Relapsed or refractory pediatric B-Cell ALL (second or greater relapse, relapse after allogeneic SCT, or persistent MRD positive disease)
  • Relapsed or refractory pediatric B-cell non-Hodgkin's Lymphoma refractory to second-line or later chemotherapy
  • CD19 tumor expression demonstrated by flow cytometry or biopsy
  • Age 0 to 30 years at initial diagnosis
  • Karnofsky or Lansky performance status ≥50
  • eGFR ≥60 mL/min, bilirubin ≤2 mg/dl, ALT/AST ≤5x ULN, LVEF ≥40%
  • CNS-3 disease allowed if stabilized for at least 1 month prior to infusion
  • Patients ineligible for HSCT or with Ph+ ALL failing 3 TKI lines are eligible

Key Exclusion

  • Active untreated infection or uncontrolled infection at screening
  • Acute/ongoing neurologic toxicity >Grade 1
  • Concomitant genetic syndrome (Fanconi anemia, Kostmann, Shwachman) except Down Syndrome
  • Grade 2-4 acute or extensive chronic GVHD at enrollment
  • Active or latent hepatitis B, active hepatitis C, or HIV positive
  • Allogeneic HSCT within 3 months of enrollment
  • Any prior CD19 CAR T cell therapy
  • Pregnant or nursing women

NCT07213804

FRAmework-01: A Two-Part Phase 3 Study of LY4170156 Versus Chemotherapy or Mirvetuximab Soravtansine in Platinum-Resistant Ovarian Cancer, and LY4170156 Plus Bevacizumab Versus Platinum-Based Chemotherapy Plus Bevacizumab in Platinum-Sensitive Ovarian Cancer. Genomic-based

Organization/Sponsor: Eli Lilly and Company


Example patient: A 58-year-old woman with high-grade serous ovarian cancer, ECOG status 1, who progressed 4 months after completing second-line platinum-based chemotherapy and prior bevacizumab treatment, with known BRCA mutation and prior PARPi therapy.

Phase 3

Interventions

  • Drug: LY4170156
    Summary: LY4170156 is an antibody-drug conjugate targeting folate receptor alpha (FRα), linked to a topoisomerase I inhibitor payload, being studied for solid tumors with promising anti-tumor activity (Web Search).
  • Drug: Paclitaxel
    Summary: Paclitaxel is a microtubule inhibitor that stabilizes microtubules to prevent cancer cell division, indicated for advanced ovarian, breast, lung cancers, and Kaposi's sarcoma (FDA label, NCI Thesaurus).
  • Drug: Topotecan
    Summary: Topotecan is a topoisomerase I inhibitor that stabilizes DNA-enzyme complexes during replication, causing DNA breaks, indicated for small cell lung cancer and cervical carcinoma (FDA label, NCI Thesaurus).
  • Drug: Gemcitabine
    Summary: Gemcitabine is a nucleoside analogue that inhibits DNA synthesis by competing with deoxycytidine and inhibiting ribonucleotide reductase, indicated for pancreatic, lung, breast, and ovarian cancers (FDA label, NCI Thesaurus).
  • Drug: Pegylated liposomal doxorubicin
    Summary: Pegylated liposomal doxorubicin is an encapsulated anthracycline chemotherapy that targets cancer cells while reducing toxicity to normal tissues, used for refractory solid tumors (Web Search).
  • Drug: MIRV
    Summary: Mirvetuximab soravtansine (MIRV) is an antibody-drug conjugate targeting folate receptor alpha, delivering a cytotoxic payload, FDA-approved in 2023 for platinum-resistant ovarian cancer (Web Search).
  • Biological: Bevacizumab
    Summary: Bevacizumab is a monoclonal antibody that blocks vascular endothelial growth factor (VEGF) to inhibit tumor angiogenesis, used in various cancers including ovarian cancer (Web Search).
  • Drug: Carboplatin
    Summary: Carboplatin is a platinum-based agent that forms DNA cross-links to induce apoptosis and inhibit cell growth, indicated for advanced and recurrent ovarian carcinoma (FDA label, NCI Thesaurus).

Key Inclusion

  • Histologically confirmed high-grade serous ovarian, primary peritoneal, or fallopian tube cancer
  • Radiographic progression on or after most recent systemic anticancer therapy
  • ECOG performance status 0-1
  • Measurable disease per RECIST v1.1
  • Part A: Platinum-resistant disease (progression ≤6 months after last platinum)
  • Part A: 1-3 prior lines of systemic cytotoxic therapy (up to 4 if one is mirvetuximab)
  • Part B: Platinum-sensitive disease (progression >6 months after last platinum)
  • Part B: Prior PARPi with progression on or within 6 months of completion

Key Exclusion

  • Prior antibody-drug conjugate with topoisomerase inhibitor payload
  • Part A: Primary platinum-refractory disease (progression ≤3 months since first-line platinum)
  • Part B: Clinically significant proteinuria

NCT07109219

A Modular Phase I/II, Open-label, Multi-center Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Preliminary Efficacy of AZD4512 Monotherapy or in Combination With Anticancer Agent(s) in Participants With Acute Lymphoblastic Leukemia Genomic-based

Organization/Sponsor: AstraZeneca


Example patient: A 14-year-old with CD22-positive Philadelphia chromosome-negative B-ALL who relapsed after two prior chemotherapy regimens, has bone marrow blasts of 15%, ECOG performance status of 1, and no active CNS disease.

Phase 1, Phase 2

Interventions

  • Drug: AZD4512 monotherapy
    Summary: AZD4512 is a targeted therapy for CD22-positive B-cell acute lymphoblastic leukemia that targets specific molecular pathways; ongoing clinical trials assess its efficacy and safety in relapsed/refractory disease (Source: Web Search).

Key Inclusion

  • Age 12-16 years or older depending on module
  • CD22-positive B-ALL relapsed or refractory with bone marrow blasts >5%
  • ECOG ≤2, KPS ≥50, or LPS ≥50
  • Peripheral lymphoblast count <10,000/µL
  • At least 2 prior therapies with relapse/refractoriness or 1 prior therapy with no standard options
  • Prior CD22 targeted therapies allowed
  • Ph+ B-ALL must be TKI intolerant or refractory to at least 2 TKIs
  • Adequate washout from prior cell therapy, DLI, or transplant

Key Exclusion

  • Burkitt lymphoma or leukemia
  • Isolated extramedullary disease or active testicular/CNS involvement (>CNS1)
  • Unresolved non-heme toxicities Grade ≥2
  • History of drug-induced ILD/pneumonitis requiring steroids or oxygen
  • Cytotoxic treatment within 14 days except maintenance or cytoreduction
  • Biologic treatment within 28 days or 5 half-lives
  • Strong CYP3A4 inhibitors or QTc-prolonging medications within specified washout
  • Investigational agents within 30 days or 5 half-lives

NCT07194044

Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes Genomic-based

Organization/Sponsor: H. Lee Moffitt Cancer Center and Research Institute


Example patient: A 14-year-old male with newly diagnosed widely metastatic Ewing sarcoma involving bone and bone marrow, who has received one cycle of VDC chemotherapy and has adequate organ function with tissue available for molecular testing.

Phase N/A

Interventions

  • Drug: Liposomal doxorubicin
    Summary: Liposomal doxorubicin damages DNA and blocks enzymes needed for cell division, used in treating sarcomas and various cancers (NCI Thesaurus, Web Search).
  • Drug: Etoposide
    Summary: Etoposide inhibits topoisomerase II, disrupting DNA replication and transcription, leading to apoptotic cell death, primarily acting in G2 and S phases (FDA label, NCI Thesaurus).
  • Drug: Temozolomide
    Summary: Temozolomide is an alkylating agent that methylates DNA at O6 and N7 positions of guanine, inhibiting DNA replication, with good CNS penetration (FDA label, NCI Thesaurus).
  • Drug: Topotecan
    Summary: Topotecan inhibits topoisomerase I by stabilizing topoisomerase I-DNA complexes, producing potentially lethal double-strand DNA breaks during replication (FDA label, NCI Thesaurus).
  • Drug: Cabozantinib
    Summary: Cabozantinib inhibits multiple receptor tyrosine kinases including MET, RET, and VEGFRs, inhibiting tumor growth and angiogenesis (FDA label, NCI Thesaurus).
  • Drug: Irinotecan
    Summary: Irinotecan is a prodrug converted to SN-38, which inhibits topoisomerase I, stabilizing DNA-enzyme complexes and triggering apoptosis as an S-phase-specific agent (FDA label, NCI Thesaurus).
  • Drug: Actinomycin
    Summary: Actinomycin (dactinomycin) intercalates into DNA and inhibits topoisomerase II, blocking RNA and protein synthesis, used in pediatric sarcomas and Wilms tumor (FDA label, NCI Thesaurus).
  • Drug: Ifosfamide
    Summary: Ifosfamide is an alkylating agent that forms DNA crosslinks, preventing DNA replication; requires activation by hepatic enzymes and mesna for bladder protection (FDA label, NCI Thesaurus).
  • Drug: Cyclophosphamide
    Summary: Cyclophosphamide is converted to active metabolites that alkylate DNA, inhibiting replication and initiating cell death, used in various malignancies and immunosuppression (FDA label, NCI Thesaurus).
  • Drug: Doxorubicin
    Summary: Doxorubicin intercalates DNA and inhibits topoisomerase II, preventing DNA replication and forming oxygen free radicals, with dose-dependent cardiotoxicity risk (FDA label, NCI Thesaurus).
  • Drug: Vincristine
    Summary: Vincristine binds microtubules and spindle proteins, arresting tumor cells in metaphase by interfering with mitotic spindle formation (FDA label, NCI Thesaurus).

Key Inclusion

  • Patients must be >1 year of age with no upper age limit
  • New histologic diagnosis of widely metastatic Ewing sarcoma or metastatic CIC-rearranged sarcoma
  • Sufficient tissue submitted for correlative testing (flash frozen, FFPE block, or unstained slides)
  • May have started initial cycle of VDC prior to enrollment but not IE
  • Adequate organ function
  • Healthy enough to tolerate protocol therapy
  • Use of two methods of contraception or abstinence for reproductive-age patients
  • Written informed consent or assent

Key Exclusion

  • Localized disease or lung-only metastases for Ewing sarcoma
  • Central nervous system tumors (primary or metastatic)
  • Receiving other investigational agents for cancer
  • History of cancer treated with myelosuppressive chemotherapy or radiation
  • Receiving additional medicines for treating cancer
  • Uncontrolled intercurrent illness
  • Pregnancy or breastfeeding
  • Unable to comply with safety monitoring requirements

NCT06995872

Phase I Trial of rhIL-15 Plus Dinutuximab Plus Irinotecan/Temozolomide for Children and Young Adults With Relapsed/Refractory Neuroblastoma

Organization/Sponsor: National Institutes of Health Clinical Center (CC)


Example patient: A 7-year-old with relapsed neuroblastoma after frontline chemotherapy and dinutuximab salvage therapy, with MIBG-avid bone lesions, Lansky score 60%, adequate organ function, and no active infection.

Phase I

Interventions

  • Drug: Irinotecan Hydrochloride
    Summary: Topoisomerase I inhibitor that stabilizes the cleavable complex between topoisomerase I and DNA, causing DNA breaks that inhibit replication and trigger apoptosis; S-phase-specific agent used in neuroblastoma and other pediatric cancers (NCI Thesaurus, FDA label).
  • Drug: Temozolomide
    Summary: Alkylating agent that converts to MTIC at physiologic pH, methylating DNA at O6 and N7 positions of guanine to inhibit DNA replication; penetrates CNS well and used in pediatric brain tumors and neuroblastoma (NCI Thesaurus, FDA label).
  • Biological: Dinutuximab
    Summary: Chimeric monoclonal antibody that binds to GD2 ganglioside on tumor cells, inducing antibody-dependent cell-mediated cytotoxicity and complement-dependent cytotoxicity; FDA-approved for high-risk neuroblastoma in combination with immunostimulatory agents (NCI Thesaurus, FDA label).
  • Biological: rhIL-15
    Summary: Recombinant human interleukin-15 that regulates T and NK cell activation and proliferation via JAK-STAT signaling pathways; enhances anti-tumor immune activity and CD8+ memory T cell expansion in cancer immunotherapy (NCI Thesaurus).

Key Inclusion

  • Pathology-confirmed neuroblastoma with relapsed/refractory disease after at least 4 cycles frontline high-risk chemotherapy and one salvage treatment
  • Evaluable disease by MIBG, PET, MRI/CT, or bone marrow with measurable tumors ≥10 mm or MIBG-avid lesions
  • Age ≥3 years and ≤35 years at enrollment
  • Karnofsky ≥50% (≥16 years) or Lansky ≥50% (<16 years)
  • ANC ≥1000/mcL, platelets ≥100,000/mcL transfusion-independent
  • Left ventricular ejection fraction ≥52%
  • Oxygen saturation >92% on room air at rest
  • Adequate renal and hepatic function with creatinine clearance ≥60 mL/min/1.73 m² and AST/ALT ≤3x ULN

Key Exclusion

  • Presence of pericardial effusion
  • Current/active HIV, HBV, or HCV infection
  • History of severe hypersensitivity to irinotecan or temozolomide
  • Grade ≥2 diarrhea at entry
  • Uncontrolled infection or intercurrent illness
  • Positive pregnancy test or nursing
  • History of Grade 4 allergic reactions to anti-GD2 antibodies requiring permanent discontinuation
  • Unable to tolerate oral/nasogastric/gastrostomy medications or significant malabsorption

NCT06492954

Phase 1b Trial of Atezolizumab in Combination With Stereotactic Body Radiation Therapy (SBRT) and Surgery in Patients With Pulmonary Recurrence of Osteosarcoma

Organization/Sponsor: Emory University


Example patient: A 14-year-old with second relapse osteosarcoma presenting with three resectable bilateral lung nodules (largest 8 mm), Lansky score 80, adequate organ function, and no extrapulmonary disease.

Phase 1

Interventions

  • Biological: Atezolizumab
    Summary: Atezolizumab is a humanized PD-L1 blocking monoclonal antibody that enhances T-cell-mediated immune response by preventing PD-L1 binding to PD-1 and B7.1, thereby reversing T-cell inactivation and promoting antineoplastic activity. FDA-approved for multiple cancers including NSCLC and melanoma, it is being studied in pediatric solid tumors like osteosarcoma. Source: FDA label, NCI Thesaurus.
  • Radiation: Stereotactic Body Radiation Therapy (SBRT)
    Summary: SBRT delivers high-dose, precise radiation to body tumors in one or several treatments, minimizing damage to surrounding tissues. Used in pediatric sarcomas and advanced cancers to target localized metastatic lesions. Source: NCI Thesaurus, Web Search.
  • Procedure: Surgical Resection
    Summary: Surgical removal of all or part of a tumor or tissue structure. Used in pediatric cancer when tumors are localized and resectable, aiming for complete removal of cancerous growths. Source: NCI Thesaurus.

Key Inclusion

  • First or greater relapse of osteosarcoma with histologic verification
  • Recurrence limited to lung (unilateral or bilateral)
  • All pulmonary nodules resectable without pneumonectomy
  • At least 1 lesion ≥5 mm eligible for SBRT plus additional nodule(s) requiring resection
  • Lansky/Karnofsky score ≥60 or ECOG ≤2
  • Adequate organ function: ANC ≥750/mm3, platelets ≥50,000/mm3, creatinine clearance ≥70 ml/min/1.73m2
  • Life expectancy at least 4 months
  • Effective contraception required for sexually active patients of reproductive potential

Key Exclusion

  • Active metastatic disease outside lungs (bone, CNS, extrapulmonary)
  • Prior lung radiation
  • Active autoimmune disorder requiring systemic treatment in past 12 months
  • Significant cardiovascular disease within 3 months
  • Prior allogeneic stem cell or solid organ transplant
  • Chronic immunosuppressive therapies or uncontrolled infection
  • Known HIV, hepatitis B/C, or current/prior pneumonitis
  • Pregnancy or breastfeeding

NCT06816979

Assessing the CSF-ctDNA of Patients With Stage III and IV Non-Small Cell Lung Cancer: A Pilot Study Genomic-based

Organization/Sponsor: Ohio State University Comprehensive Cancer Center


Example patient: A 58-year-old male with newly diagnosed stage IV NSCLC harboring an EGFR mutation, estimated survival over one year, receiving systemic therapy at Ohio State University, with no prior cancer history or contraindications to MRI or lumbar puncture.

Phase N/A

Interventions

  • Procedure: Magnetic Resonance Imaging
    Summary: MRI uses radiofrequency waves and magnetic fields to visualize tumors, monitor treatment effects, and assess tumor size and relation to surrounding tissues (NCI Thesaurus, Web Search).
  • Procedure: Lumbar Puncture
    Summary: Invasive procedure inserting a hollow needle through the lower back to access cerebrospinal fluid for cancer diagnosis and sampling (NCI Thesaurus, Web Search).
  • Procedure: Biospecimen Collection
    Summary: Collection of tissue or fluid samples for testing and research to study genetic features and identify disease patterns in cancer (NCI Thesaurus, Web Search).

Key Inclusion

  • New histological diagnosis of stage III or IV NSCLC
  • Documented mutation on lung cancer mutation panel (PULMOL) for stage III/IV without brain metastases
  • Patient treated with radiation therapy and/or systemic therapy at Ohio State University
  • Estimated survival >= 1 year
  • No medical contraindication to lumbar puncture

Key Exclusion

  • Alzheimer's, dementia, or mental disability
  • Not able to receive MRI
  • Allergy to xylocaine or any numbing medication for lumbar puncture
  • Previous cancer history prior to diagnosis of NSCLC
  • Pregnant or lactating women
  • Women of childbearing potential or sexually active men not using medically acceptable contraception

NCT07059975

UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML: A Pilot Trial Genomic-based

Organization/Sponsor: Baylor College of Medicine


Example patient: A 7-year-old with newly diagnosed AML harboring RUNX1::RUNX1T1 translocation, Lansky score 80, normal cardiac and renal function, who completed DA10+GO induction therapy.

Phase N/A

Interventions

  • Standard of Care: SOC
    Summary: Standard of care refers to conventional therapies including chemotherapy and radiation, working through cell cycle disruption and DNA damage (Summary of Web Search, NCI Thesaurus).
  • Drug: Intrathecal triple
    Summary: Combination of methotrexate, cytarabine, and hydrocortisone delivered intrathecally to prevent CNS relapse in high-risk leukemia by direct CNS delivery (Summary of Web Search, NCI Thesaurus).
  • Biological: Asparaginase Erwinia Chrysanthemi (recombinant)
    Summary: Recombinant bacterial enzyme that depletes asparagine, essential for leukemic cell survival, indicated for ALL and LBL in patients with E. coli-derived asparaginase hypersensitivity (FDA label, NCI Thesaurus).
  • Drug: Etoposide
    Summary: Topoisomerase II inhibitor that prevents DNA replication and transcription, used in combination therapy for pediatric leukemia and lymphoma, acting primarily in G2 and S phases (FDA label, NCI Thesaurus).
  • Drug: Venetoclax
    Summary: Selective BCL-2 inhibitor that restores apoptosis in cancer cells by binding BCL-2 proteins, indicated for CLL, SLL, and AML in combination with hypomethylating agents (FDA label, NCI Thesaurus).
  • Drug: Cytarabine (Ara-C)
    Summary: Nucleoside metabolic inhibitor that disrupts DNA synthesis by incorporating into DNA, used to treat acute leukemias via IV, subcutaneous, or intrathecal routes (FDA label, Summary of Web Search).
  • Drug: Fludarabine
    Summary: Fluorinated nucleotide analog that inhibits DNA polymerase, ribonucleotide reductase, and DNA primase, interrupting DNA synthesis in B-cell CLL and other leukemias (FDA label, NCI Thesaurus).
  • Drug: Idarubicin Hydrochloride
    Summary: Anthracycline antibiotic that intercalates DNA and inhibits topoisomerase II, indicated for AML in combination therapy, with high lipophilicity enabling efficient cell membrane penetration (FDA label, NCI Thesaurus).

Key Inclusion

  • Age 1 month to 30 years
  • AML or myeloid sarcoma per 2022 WHO classification
  • ≥20% bone marrow blasts or specific genetic abnormalities
  • Must receive DA10+GO induction therapy
  • Karnofsky/Lansky performance status >40
  • Creatinine clearance ≥60 ml/min/1.73m2
  • Ejection fraction ≥50% or shortening fraction ≥24%
  • Direct bilirubin <2 mg/dL and ALT <5x ULN

Key Exclusion

  • Constitutional conditions: Fanconi anemia, Schwachman Diamond Syndrome, telomere disorders, trisomy 21
  • Therapy-related AML
  • Acute promyelocytic leukemia
  • AML with FLT3-ITD
  • Mixed phenotype acute leukemia
  • Philadelphia chromosome positive AML
  • Pregnancy or breastfeeding
  • Concurrent malignancy or juvenile myelomonocytic leukemia

NCT06176690

Constitutive IL7R (C7R) Modified Banked Allogeneic CD30 Chimeric Antigen Receptor Epstein-Barr Virus-Specific T Lymphocytes (CD30.CAR-EBVSTs) in Patients With Relapsed or Refractory CD30-Positive Lymphomas Genomic-based

Organization/Sponsor: Baylor College of Medicine


Example patient: A 16-year-old with relapsed Hodgkin lymphoma confirmed CD30-positive by CLIA laboratory, Karnofsky score 70%, normal renal and hepatic function, no active infections, and not on high-dose steroids.

Phase N/A

Interventions

  • Biological: C7R.CD30.CAR-EBVST cells
    Summary: Allogeneic EBV-specific T cells engineered with a CAR targeting CD30 and modified with constitutive IL7R. They recognize and lyse CD30-expressing tumor cells via CAR binding, causing tumor cell death in CD30-positive lymphomas. Source: NCI Thesaurus and Web Search.

Key Inclusion

  • CD30-positive tumor confirmed in CLIA certified laboratory
  • Hodgkin lymphoma, CD30+ aggressive B-cell lymphoma, or peripheral T-cell lymphoma
  • Age 12 to 75 years
  • Bilirubin ≤2x upper limit of normal (≤3x for Gilbert syndrome)
  • Estimated GFR >70 mL/min
  • Karnofsky or Lansky score >60%
  • Recovered from acute non-hematologic chemotherapy toxicities
  • Effective birth control during and 6 months after study

Key Exclusion

  • Investigational cell therapy or vaccine within past 6 weeks
  • CD30 antibody-based therapy within previous 4 weeks
  • Hypersensitivity to murine protein-containing products
  • Pregnant or lactating
  • Tumor location risking airway obstruction upon enlargement
  • Systemic corticosteroids ≥10 mg/day prednisone equivalent
  • Active uncontrolled bacterial, viral, or fungal infection
  • Symptomatic cardiac disease (NYHA Class III or IV)

NCT07223021

Improving EveNt Free Survival by Optimizing FLUdarabine Exposure During LymphodepletioN for CAR T CEll Therapy: a Randomized, Multi-center Study of Children and Young Adults With B-cell Acute Lymphoblastic Leukemia (INFLUENCE)

Organization/Sponsor: Memorial Sloan Kettering Cancer Center


Example patient: A 12-year-old child weighing 35 kg with relapsed B-cell acute lymphoblastic leukemia, Lansky score 70%, normal cardiac and renal function, eligible for commercial tisagenlecleucel therapy.

Phase N/A

Interventions

  • Biological: CAR-T (Tisagenlecleucel)
    Summary: Engineered T-cells targeting CD19 on B-cell malignancies, inducing immune-mediated cancer cell death in relapsed/refractory leukemia and lymphoma (Summary of Web Search).
  • Drug: Cyclophosphamide
    Summary: Alkylating agent that binds DNA to inhibit replication, used for lymphodepletion before CAR-T therapy and treating leukemias, lymphomas, and solid tumors (FDA label, NCI Thesaurus).
  • Drug: Fludarabine
    Summary: Fluorinated nucleoside analog inhibiting DNA synthesis via fludarabine triphosphate, used for lymphodepletion and treating chronic lymphocytic leukemia (FDA label, NCI Thesaurus).

Key Inclusion

  • B-ALL eligible for commercial tisagenlecleucel
  • Weight >9 kg at lymphodepletion
  • Serum bilirubin ≤2 mg/dL
  • GFR ≥70 ml/min/1.73m²
  • LVEF ≥50% by MUGA or echocardiogram
  • Oxygen saturation ≥90% on room air
  • ECOG ≤1 or Karnofsky >60% (age ≥16 years)
  • Lansky ≥60% (age <16 years)

Key Exclusion

  • Hypersensitivity to fludarabine, cyclophosphamide, or tisagenlecleucel
  • Out of specification tisagenlecleucel product
  • Clinically significant active uncontrolled infection
  • Unable to give informed consent or comply with protocol
  • Pregnant or lactating women

NCT07173205

R00 Full Factorial Trial: Optimizing ASCENT

Organization/Sponsor: East Carolina University


Example patient: A 22-year-old emerging adult diagnosed with leukemia at age 19, completed treatment 18 months ago, fluent in English with a smartphone, experiencing mild depression but no severe mental illness or suicidal ideation.

Phase N/A

Interventions

  • Behavioral: AYA Survivors Coping and Emotional Needs Toolkit (ASCENT)
    Summary: ASCENT is a digital intervention providing psychoeducation and evidence-based components to help adolescent and young adult cancer survivors manage depression and address emotional and coping needs (Web Search).

Key Inclusion

  • Age at enrollment 15-39 years
  • Age at diagnosis 12-39 years
  • 1 month to 5 years post-treatment completion
  • Fluent in English spoken and written
  • Own smartphone with data plan

Key Exclusion

  • Current diagnosis of severe or persistent mental illness
  • Severe suicidal ideation including plan and intent

NCT07218848

ProSalud: A Health Promotion Project to Reduce Cancer Risk in Latinos Living in Rural/Agricultural Communities Within Moffitt's Catchment Area

Organization/Sponsor: H. Lee Moffitt Cancer Center and Research Institute


Example patient: A 35-year-old Spanish-speaking mother of an 8-year-old child living in a rural agricultural community seeking cancer prevention education.

Phase N/A

Interventions

  • Behavioral: Aim 2 Focus Groups
    Summary: Qualitative focus groups with families and healthcare providers to gather feedback and refine psychosocial support interventions for cancer risk reduction (NCI Thesaurus, Web Search).
  • Behavioral: Aim 1 Workshops
    Summary: Educational workshops designed to develop targeted health promotion interventions to reduce cancer risk through supportive and educational strategies (Web Search).

Key Inclusion

  • Adults of any age and sex who are parents
  • Parent of a 6- to 12-year-old child
  • Must speak Spanish
  • Able to read and write at 4th-grade level
  • Parents who speak English in addition to Spanish can participate
  • Workshop conducted in Spanish

Key Exclusion

  • Individuals who are wards of the state

NCT07172958

Selective Antigen Specific dTβRII-expressing T Cells and B7-H3 CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)

Organization/Sponsor: Children's National Research Institute


Example patient: A 7-year-old child weighing 25 kg with relapsed Ewing sarcoma after standard chemotherapy, Karnofsky score of 70, adequate organ function, no CNS involvement, and no recent steroid use.

Phase N/A

Interventions

  • Biological: Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
    Summary: Engineered T cells targeting B7-H3 antigen on cancer cells combined with dTβRII-expressing T cells for pediatric solid tumors. Mechanism involves CAR T cell recognition and attack of B7-H3-expressing tumor cells. Source: Web Search Summary.

Key Inclusion

  • Relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor
  • Age ≥1 year and <24 years
  • Weight >10 kg
  • Karnofsky/Lansky score ≥60
  • Measurable or evaluable disease by imaging
  • ANC >500/µL for procurement, >750/µL for infusion
  • No systemic steroids within 1 week
  • Adequate cardiac function with shortening fraction ≥27% or ejection fraction >50%

Key Exclusion

  • Known CNS disease
  • Uncontrolled infections or known HIV infection
  • Pregnant or lactating females
  • Previous allogeneic stem cell transplant
  • Whole lung/mediastinal radiation within 12 weeks of infusion
  • Clinically significant systemic illness interfering with safety assessment

NCT06814795

Feasibility of Medically Tailored Meals for Pediatric Populations at Risk for Disparities in Serious Illness Outcomes Due to Inequities in Social Drivers of Health (MTM-Kids)

Organization/Sponsor: UNC Lineberger Comprehensive Cancer Center


Example patient: A 14-year-old Spanish-speaking patient receiving ongoing chemotherapy for leukemia who has completed one cycle and experiences taste changes, with a parent participating as caregiver.

Phase N/A

Interventions

  • Dietary Intervention: Medically tailored meals
    Summary: Customized nutrition interventions providing nutritious meals aligned with medical needs to improve dietary intake and reduce treatment-related anxiety in pediatric cancer patients (Source: Web Search).

Key Inclusion

  • Under care of UNC Chapel Hill Pediatric Hematology/Oncology
  • Completed at least one cycle of chemotherapy with taste-altering agents
  • Expect to undergo at least two more cycles
  • Aged 12-17.9 years
  • Communicate in English or Spanish
  • Primary parental caregiver aged 18 years or older

Key Exclusion

  • None if inclusion criteria are met