Sophic Logo gordian knotPediatric Cancer Clinical Trials Intelligence

Monthly Update Report for Trials Started in May 2026


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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 leukemia, lymphoma, brain tumors, neuroblastoma, sarcomas, and breast cancer. Interventions range from supportive care (exercise, cognitive training, family-based weight management) to experimental therapies (CAR-T cells, gene therapy, targeted inhibitors, RNA-lipid vaccines). Several trials focus on relapsed/refractory disease, novel immunotherapies, and biomarker-driven treatments. Prevention studies address HPV vaccination, lung health in smokers, and ototoxicity mitigation. Many trials emphasize pediatric populations under 25-39 years, with some extending to adults for specific tumor types.

Common Criteria Across Trials

Common Inclusion

  • Age ranges typically 0-39 years, with some trials extending to adults
  • Histologically confirmed cancer diagnosis or relapsed/refractory disease
  • Adequate organ function (liver, kidney, cardiac, bone marrow)
  • Performance status (Karnofsky/Lansky ≥50-60, ECOG ≤2)
  • Informed consent from parent/guardian or patient
  • Recovery from prior therapy with specified washout periods
  • Measurable or evaluable disease per RAPNO or other criteria
  • Ability to comply with study procedures and follow-up

Common Exclusion

  • Uncontrolled infection or active HIV/hepatitis
  • Pregnancy or breastfeeding
  • Severe cardiac, renal, pulmonary, or hepatic dysfunction
  • Active GVHD requiring systemic therapy
  • Concurrent investigational agents or other anti-cancer therapy
  • Severe psychiatric illness or cognitive impairment
  • Hypersensitivity to study agents
  • CNS disease (progressive or symptomatic in some trials)
  • Recent chemotherapy, radiation, or transplant within specified timeframes

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


NCT07474090

Supportive Tailored Exercise Program for Survivors of Breast Cancer (STEPS-BC)

Organization/Sponsor: Wake Forest University Health Sciences


Example patient: A 52-year-old woman with newly diagnosed Stage II ER-positive breast cancer, ECOG status 1, scheduled to start adjuvant chemotherapy, who walks daily but exercises less than 300 minutes weekly.

Phase N/A

Interventions

  • Procedure: Cardiopulmonary Exercise Testing
    Summary: Evaluates respiratory and cardiovascular responses during exercise to assess fitness and cardiac function under stress, used to determine if patients can tolerate surgical stress (NCI Thesaurus, Web Search).
  • Other: Electronic Health Record Review
    Summary: Analyzes patient data to improve breast cancer care, targeting physical activity and quality of life through personalized feedback and support (NCI Thesaurus, Web Search).
  • Procedure: Accelerometry
    Summary: Measures physical activity levels using instruments that measure acceleration, showing inverse association with breast cancer risk, particularly in postmenopausal women (NCI Thesaurus, Web Search).
  • Other: Questionnaire Administration
    Summary: Collects patient self-reported outcomes to assess quality of life and psychological and physical impacts of breast cancer treatment (NCI Thesaurus, Web Search).
  • Procedure: Magnetic Resonance Imaging
    Summary: Noninvasive imaging using radiofrequency waves and magnetic fields to provide detailed breast tissue images for detecting tumors and monitoring treatment response (NCI Thesaurus, Web Search).
  • Procedure: Biospecimen Collection
    Summary: Gathers tissue and fluid samples for testing and research to analyze genetic and molecular features of breast cancer (NCI Thesaurus, Web Search).
  • Other: Supportive Care
    Summary: Helps patients cope with cancer and treatment through symptom management, psychological support, and practical advice to maximize treatment benefits and quality of life (NCI Thesaurus, Web Search).
  • Behavioral: Educational Intervention
    Summary: Educational activity to improve knowledge and beliefs about breast cancer, addressing physical, emotional, and lifestyle changes through lectures, brochures, and multimedia (NCI Thesaurus, Web Search).
  • Behavioral: Exercise Intervention
    Summary: Managed physical activity program to improve health and wellbeing, targeting cancer-related fatigue and quality of life through modulation of tumor microenvironment (NCI Thesaurus, Web Search).

Key Inclusion

  • Stage I-III breast cancer treated with curative intent
  • Scheduled to receive neoadjuvant or adjuvant cytotoxic chemotherapy
  • Age 18 to 85 years at enrollment
  • ECOG performance status ≤ 2
  • Able to complete stationary bicycle exercise test
  • Able to walk at least 2 blocks without symptoms
  • Must have access to teleconferencing device
  • Must read and understand English language

Key Exclusion

  • Metastatic breast cancer
  • Symptomatic claustrophobia or MRI-incompatible devices
  • Pregnancy or breast-feeding
  • Uncontrolled hypertension (systolic >190 or diastolic >100 mmHg)
  • Cardiac conditions including unstable angina, heart failure, severe valvular disease
  • Acute myocardial infarction within 28 days
  • Acute pulmonary embolus or DVT within 24 weeks
  • Currently engaged in ≥300 minutes moderate to vigorous physical activity per week

NCT07558473

Measuring Brain Changes Following Cognitive Intervention in Pediatric Patients With Brain Tumors

Organization/Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Example patient: A 7-year-old child with medulloblastoma diagnosed 2 years ago, currently receiving follow-up care at Johns Hopkins Hospital, with stable disease and cognitive deficits in processing speed but able to understand and participate in computerized training exercises.

Phase N/A

Interventions

  • Behavioral: Online computerized cognitive training intervention focused on processing speed
    Summary: Online computerized cognitive training improves processing speed, attention, and working memory through task-based exercises targeting neuroplasticity and cognitive reserve enhancement in pediatric cancer survivors (Summary of Web Search).

Key Inclusion

  • Primary tumor located in the brain
  • Diagnosed between 0 and 25 years of age
  • Within five years of cancer diagnosis
  • Receiving care at Johns Hopkins Hospital

Key Exclusion

  • Primary tumor outside of the brain
  • Patients too young to participate (<5 years)
  • Severe brain injuries precluding understanding and participation in cognitive intervention

NCT06528691

PHASE 2 Study of Entrectinib as a Single Agent in Upfront Therapy for Children <3 Years of Age With NTRK1/2/3 or ROS1-FUSED CNS Tumors (GLOBOTRK) Genomic-based

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


Example patient: A 2-year-old with newly diagnosed supratentorial high-grade glioma harboring NTRK2 fusion, confirmed by central pathology 3 weeks post-biopsy, with stable neurologic deficits, adequate organ function, and no prior cancer treatment.

Phase 2

Interventions

  • Procedure: Surgery
    Summary: Surgical resection involves physical excision of cancerous tumors and surrounding tissue to eliminate malignant cells, often used in combination with other treatments (NCI Thesaurus).
  • Biological: Pegfilgrastim
    Summary: Pegfilgrastim is a recombinant granulocyte colony-stimulating factor that stimulates bone marrow to produce neutrophils, reducing infection risk in chemotherapy-induced neutropenia (FDA label).
  • Biological: G-CSF
    Summary: Filgrastim stimulates bone marrow neutrophil production to prevent febrile neutropenia in pediatric cancer patients undergoing chemotherapy (Summary of Web Search).
  • Drug: Carboplatin
    Summary: Carboplatin is a platinum-based agent that forms DNA cross-links, inducing apoptosis and inhibiting cancer cell growth, with similar efficacy to cisplatin but reduced toxicity (FDA label, NCI Thesaurus).
  • Drug: Etoposide
    Summary: Etoposide inhibits topoisomerase II, preventing DNA replication and transcription, leading to apoptotic cell death in cancer cells (FDA label, NCI Thesaurus).
  • Drug: Cyclophosphamide
    Summary: Cyclophosphamide is an alkylating agent that cross-links DNA, disrupting replication and inducing cell death in various malignancies including pediatric tumors (FDA label, NCI Thesaurus).
  • Drug: Entrectinib
    Summary: Entrectinib inhibits TrkA/B/C, ROS1, and ALK tyrosine kinases, disrupting oncogenic signaling and inducing apoptosis in NTRK or ROS1 fusion-positive tumors (FDA label, NCI Thesaurus).

Key Inclusion

  • Age <3 years at diagnosis
  • NTRK1/2/3 or ROS1 gene fusions confirmed by central pathology
  • Measurable disease by RAPNO criteria
  • No prior chemotherapy, targeted therapy, immunotherapy, cellular therapy, or radiation
  • Lansky score ≥50% and life expectancy ≥12 weeks
  • Adequate organ function (hematologic, hepatic, renal, cardiac)
  • QTc ≤450 msec and LVEF >50%
  • Neurologic deficits stable for ≥7 days

Key Exclusion

  • Prior cancer therapy (chemotherapy, targeted therapy, immunotherapy, cellular therapy, radiation)
  • Receiving enzyme-inducing antiepileptic drugs
  • Surgery within 2 weeks prior to enrollment
  • Active uncontrolled infection
  • Recent symptomatic congestive heart failure (3 months)
  • Gastrointestinal malabsorption disorder
  • Hypersensitivity to study drug excipients
  • Receiving concurrent investigational agents

NCT07374315

A Family-based Intervention Approach to Address Weight Gain in Pediatric Leukemia Survivors

Organization/Sponsor: Washington University School of Medicine


Example patient: A 12-year-old English-speaking patient with B-cell ALL currently on maintenance chemotherapy, able to walk briskly for five minutes, with negative SCOFF screen and a participating caregiver.

Phase N/A

Interventions

  • Educational Platform: WashU Extended Learning Canvas
    Summary: Educational platform targeting pre-leukemic conditions and employing innovative immunotherapies including CAR-T cell treatments for aggressive T cell cancers using gene editing mechanisms (Summary of Web Search).
  • Device: FitBit Ace
    Summary: Wearable activity monitor measuring steps, heart rate, sleep quality, and fitness metrics to encourage physical activity and improve outcomes in pediatric cancer patients (NCI Thesaurus, Summary of Web Search).
  • Educational Resource: NIH Educational Resources
    Summary: Educational interventions targeting leukemia and lymphoma patients through clinical trials focusing on innovative treatment approaches and targeted therapies to improve patient outcomes (Summary of Web Search).
  • Behavioral: Modified Guided Self-Help Family Intervention
    Summary: Family-based counseling intervention providing psychological support and structured guidance to improve coping strategies and emotional well-being for families dealing with leukemia or lymphoma (Summary of Web Search).

Key Inclusion

  • Diagnosis of ALL (T or B cell)
  • Receiving standard of care maintenance chemotherapy or within 6 months of concluding maintenance
  • Age 6 to 18 years at enrollment
  • Able to speak and understand English
  • Caregiver participating in study
  • Able to perform exercise for at least five minutes
  • Patients 10 and older must have negative SCOFF screen for disordered eating

Key Exclusion

  • Severe developmental delay or intellectual disabilities (e.g., Trisomy 21, severe autism)
  • Significant mental illness (active suicidal ideation, psychotic symptoms, manic episodes, severe substance use disorder)
  • SCOFF score of 2 or more for patients 10 and older

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 14-year-old with relapsed T-cell acute lymphoblastic leukemia 90 days post-allogeneic HSCT from a matched sibling donor, with 65% CD7-positive blasts, Karnofsky score of 70%, no active GVHD, and a suitable unrelated donor identified for second transplant.

Phase N/A

Interventions

  • Biological: CD7.CAR/28zeta T Cells
    Summary: Allogeneic T-lymphocytes engineered with a chimeric antigen receptor targeting CD7 antigen, linked to CD28 and CD3-zeta co-stimulatory domains, designed to recognize and kill CD7-expressing T-cell leukemia and lymphoma cells (NCI Thesaurus, Web Search).

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
  • Prior allogeneic donor available for CD7.CAR T-cell manufacture
  • Available partially-HLA matched allogeneic EBV-specific T cell line

Key Exclusion

  • Active acute GVHD >Grade II or active chronic GVHD >mild severity
  • Corticosteroids >0.5mg/kg prednisone equivalent
  • Immunosuppressive treatment for GVHD within 28 days of infusion
  • Clinically significant infection or uncontrolled viral reactivation
  • CNS-3 disease or CNS abnormalities
  • Cardiac dysfunction NYHA III or IV, LVEF <50%
  • Pregnant or lactating
  • Tumor location where enlargement could cause airway obstruction

NCT07492316

A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM), and Recurrent/Progressive Medulloblastoma (MB) Genomic-based

Organization/Sponsor: University of Florida


Example patient: A 12-year-old boy with second relapse of medulloblastoma previously treated with craniospinal radiation 6 months ago, Karnofsky score 70, adequate bone marrow and organ function, with tumor tissue collected for RNA extraction and no history of seizures or myocarditis.

Phase 1, Phase 2

Interventions

  • Biological: Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)
    Summary: An mRNA-based personalized cancer vaccine containing autologous tumor RNA and CMV pp65-flLAMP mRNA in DOTAP lipid particles that induces cytotoxic T-cell and memory T-cell responses targeting tumor antigens; flLAMP enhances MHC class II presentation promoting CD4-positive T-cell responses (NCI Thesaurus).

Key Inclusion

  • Age > 3 and ≤ 39 years
  • Histologically confirmed or suspected recurrent/progressive medulloblastoma in first or second relapse
  • Must have received radiation therapy as part of prior therapy
  • Karnofsky ≥ 60 for participants > 16 years or Lansky ≥ 60 for participants < 16 years
  • ANC ≥ 1,000/μl unsupported, platelets ≥ 100/μl unsupported for at least 7 days
  • Creatinine clearance or GFR ≥ 70mL/min/1.73 m2
  • Sterile collection of tumor material suitable for RNA extraction and loading
  • Negative pregnancy test for women of childbearing potential

Key Exclusion

  • Diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E+
  • Bulky disease with significant mass effect, uncal herniation, midline shift, or extensive multilobular involvement
  • Known HIV, Hepatitis B, or Hepatitis C seropositive
  • Uncontrolled seizure disorder
  • History of myocarditis
  • Receipt of any live vaccine within 30 days prior to enrollment
  • Pregnant or breast-feeding women
  • Prior exposure to pp65-directed therapy or any RNA-LP therapy

NCT07022678

A Randomized Double-Blinded Trial of Xylitol Dental Wipes for the Prophylaxis of Bloodstream Infections From Oral Organisms in Pediatric Patients With Acute Myeloid Leukemia

Organization/Sponsor: Children's Oncology Group


Example patient: A 7-year-old child with newly diagnosed acute myeloid leukemia without Down syndrome, planned for cytarabine-based chemotherapy with expected prolonged neutropenia, who has visible teeth and no allergies to xylitol or grapes.

Phase N/A

Interventions

  • Device/Product: Xylitol-containing Oral Wipe
    Summary: Xylitol-containing oral wipes reduce cariogenic bacteria and prevent tooth decay, used in pediatric cancer care to improve oral health and reduce infections (Summary of Web Search).
  • Other: Placebo Administration
    Summary: Medically inactive substance used as control to evaluate treatment efficacy by comparing outcomes against no active treatment (NCI Thesaurus).
  • Procedure: Electronic Health Record Review
    Summary: Checking and assessing patient data in electronic health records to monitor outcomes and improve care (NCI Thesaurus).
  • Drug: Cytarabine
    Summary: Antineoplastic nucleoside analog that inhibits DNA synthesis by competing with cytidine during S phase, used for treating acute leukemias in combination chemotherapy regimens (FDA label, NCI Thesaurus).
  • Procedure: Biospecimen Collection
    Summary: Gathering tissue and fluid samples for testing, diagnostic, and research purposes to analyze disease mechanisms (NCI Thesaurus).
  • Other: Best Practice
    Summary: Informed treatment recommendation expected to be helpful to the greatest number of people within a patient group (NCI Thesaurus).

Key Inclusion

  • Age 1 to 25 years at enrollment
  • Diagnosis of AML per 2016 WHO classification
  • Newly diagnosed or relapsed AML
  • Planned to receive at least 2 consecutive cycles of myelosuppressive chemotherapy containing IV cytarabine
  • Expected severe neutropenia duration ≥7 days per cycle
  • Minimum of one visible or erupted tooth
  • Agree to avoid xylitol-containing gum or toothpaste during intervention

Key Exclusion

  • Down syndrome-associated AML
  • Prior radiation treatment for cancer of oral cavity, head or neck in past 6 months
  • Known history of allergy to xylitol
  • Known history of allergy to grapes or grape flavoring
  • Actively being treated for oral organism related bloodstream infection
  • Unable to comply with use of oral dental wipes per practitioner assessment

NCT07553819

Effects of Respiratory Muscle Training and Aerobic Exercise on Lung Health in Smokers: A Pilot Parallel-Group Study for Lung Cancer Prevention

Organization/Sponsor: Roswell Park Cancer Institute


Example patient: A 55-year-old current smoker with 25-pack-year history, no prior cancer diagnosis, no recent respiratory infections, and stable health seeking lung cancer prevention strategies.

Phase N/A

Interventions

  • Behavioral: Respiratory Muscle Training
    Summary: Strengthens diaphragm and respiratory muscles through resistance and endurance training to improve lung function and reduce breathing difficulties. Used in lung cancer patients to minimize post-surgery fatigue and treatment side effects (NCI Thesaurus, Web Search).
  • Behavioral: Aerobic Exercise
    Summary: Sustained exercise increasing oxygen demand on cardiovascular system, enhancing cardiorespiratory fitness and potentially immune function. Improves survival rates and reduces cancer progression in lung cancer patients (NCI Thesaurus, Web Search).
  • Other: Survey Administration
    Summary: Presents questionnaires to obtain self-reported data on patient experiences, anxiety, and knowledge. Used to assess trial participation willingness and patient outcomes (NCI Thesaurus, Web Search).
  • Other: Supportive Care
    Summary: Helps patients cope with cancer effects through symptom management, pain control, and psychological support. Maximizes treatment benefits and improves quality of life throughout disease course (NCI Thesaurus, Web Search).
  • Other: Physical Performance Testing
    Summary: Assesses exercise capacity and physical function through tests like walking speed and timed activities. Evaluates fitness in lung cancer patients to guide treatment decisions (NCI Thesaurus, Web Search).
  • Other: Electronic Health Record Review
    Summary: Analyzes patient data to improve care outcomes through personalized feedback and support. Enhances patient engagement and quality of life assessment (NCI Thesaurus, Web Search).
  • Other: Educational Intervention
    Summary: Educational activity using lectures, brochures, and multimedia to prevent disease or alter disease course. Addresses physical, emotional, and lifestyle changes to improve knowledge and outcomes (NCI Thesaurus, Web Search).
  • Other: Biospecimen Collection
    Summary: Gathers tissue and fluid samples for research to analyze genetic and molecular features. Helps understand cancer mechanisms and develop targeted therapies (NCI Thesaurus, Web Search).
  • Other: Accelerometry
    Summary: Quantifies movement using acceleration measurement instruments to assess physical activity levels. Shows inverse association with cancer risk through increased activity (NCI Thesaurus, Web Search).

Key Inclusion

  • Age ≥ 50 years old
  • Current smoker with ≥ 20-pack-years history
  • Former smoker within past 15 years with ≥ 20 pack-years (CT scan cohort)
  • Able to speak, read and comprehend English
  • Cognitively capable of following direction and performing intervention
  • Signed informed consent

Key Exclusion

  • Previous lung cancer diagnosis or undergoing cancer treatment
  • Recent pneumonia, bronchitis, or inflammatory lung conditions within 6 months
  • COPD or asthma exacerbation within previous 6 months
  • Uncontrolled intercurrent illness including heart failure or psychiatric illness
  • Unwilling or unable to follow protocol requirements

NCT07471685

Development and Pilot Testing of a Family Building Decision Support Intervention for Female Adolescent and Young Adult Cancer Survivors

Organization/Sponsor: Duke University


Example patient: A 25-year-old female Hodgkin lymphoma survivor diagnosed at age 17, treated with ABVD chemotherapy and chest radiation, now in remission and considering future pregnancy options.

Phase N/A

Interventions

  • Behavioral: Survivorship Education
    Summary: Educational intervention improving health knowledge and follow-up care adherence for pediatric cancer survivors through educational programs and electronic health interventions, targeting long-term health management (Summary of Web Search).
  • Behavioral: Nurse Navigator-Delivered Family Building Decision Support
    Summary: Nurse-led support intervention providing emotional and informational guidance to female AYA cancer survivors for family-building decisions, reducing uncertainty and improving decision-making (Summary of Web Search).

Key Inclusion

  • Female AYA cancer survivor age 15-39 at diagnosis and 18-39 at enrollment
  • Completed cancer treatment with curative intent
  • Prior exposure to gonadotoxic treatments (alkylating agents, total body irradiation)
  • Prior exposure to treatments increasing obstetric risk (chest radiation, uterine radiation, anthracyclines)
  • Has not completed family building
  • Able to speak and read English
  • Willing to complete videoconference sessions and REDCap surveys

Key Exclusion

  • Visual impairment interfering with participation
  • Hearing impairment interfering with participation
  • Cognitive impairment interfering with participation
  • Severe mental illness interfering with participation

NCT07211737

(INCITE-ON) Phase I Study of i15.NKG2D.Zeta-NK Cell Conditioning in the Tumor Micro-environment in Combination With C7R.GD2.CAR-T for the Treatment of Patients With Relapsed or Refractory Osteosarcoma or Neuroblastoma Genomic-based

Organization/Sponsor: Baylor College of Medicine


Example patient: A 5-year-old with relapsed neuroblastoma after standard chemotherapy, Karnofsky score 60%, no brain metastases, adequate organ function, and available GD2.CAR-expressing autologous T-cells.

Phase I

Interventions

  • Biological: i15.NKG2D.zeta NK cells and C7R.GD2.CARTs cells
    Summary: Genetically modified immune cells targeting GD2-expressing cancers, enhancing immune cell activity and persistence through NK cell conditioning and CAR-T cell therapy. Being tested in pediatric cancer trials for safety and efficacy. Source: Summary of Web Search.

Key Inclusion

  • Neuroblastoma or Osteosarcoma with persistent, relapsed, or refractory disease after standard treatment
  • Karnofsky/Lansky score of 50% or greater for treatment
  • Greater than 1 year of age
  • Available autologous T-cells with ≥20% expressing GD2.CAR
  • Pulse Ox ≥90% on room air
  • AST <5 times upper limit of normal
  • Total bilirubin <3 times upper limit of normal
  • Serum creatinine <3 times upper limit of normal

Key Exclusion

  • History of hypersensitivity to murine protein-containing products
  • Known presence of Human Anti-Mouse Antibodies (HAMA)
  • Primary brain tumor or known brain metastases
  • Active autoimmune disease requiring immunosuppressive treatment in past 6 months
  • Tumor potentially causing airway obstruction
  • Pregnancy or lactation
  • Currently receiving immunosuppressive drugs (except low dose corticosteroids <0.25 mg/kg/day prednisone equivalent)
  • Not recovered from acute toxic effects of prior chemotherapy

NCT07447076

A Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT) Genomic-based

Organization/Sponsor: University of California, San Francisco


Example patient: A 4-year-old child with recurrent CNS atypical teratoid rhabdoid tumor confirmed by SMARCB1 loss on immunohistochemistry, Lansky score of 60, adequate bone marrow and organ function, and evaluable leptomeningeal disease on MRI.

Phase N/A

Interventions

  • Procedure: Tumor Biopsy
    Summary: Removal of tissue specimens from the living body for microscopic examination to establish diagnosis (NCI Thesaurus).
  • Drug: Paxalisib
    Summary: A PI3K inhibitor that blocks PI3K/AKT signaling pathway, inhibiting tumor cell growth and survival in susceptible populations (NCI Thesaurus).
  • Drug: Gemcitabine
    Summary: A nucleoside analog that inhibits DNA synthesis by incorporating into DNA and inhibiting ribonucleotide reductase, approved for multiple solid tumors (FDA label, NCI Thesaurus).
  • Procedure: Magnetic Resonance Imaging (MRI)
    Summary: Imaging using radiofrequency waves and magnetic fields to provide detailed pictures of internal organs, valuable for cancer diagnosis and monitoring (NCI Thesaurus).
  • Procedure: Blood Specimen Collection
    Summary: Gathering of blood samples for subsequent analysis (NCI Thesaurus).
  • Procedure: Lumber Puncture
    Summary: Invasive procedure using hollow needle through intervertebral space to access subarachnoid space for cerebrospinal fluid sampling or medication administration (NCI Thesaurus).

Key Inclusion

  • Pathologic diagnosis of CNS ATRT with SMARCB1 (INI1) loss by IHC and/or biallelic loss
  • Recurrent or progressive ATRT
  • Age ≥1 and ≤39 years at enrollment
  • Karnofsky ≥50 (>16 years) or Lansky ≥50 (≤16 years)
  • ANC ≥750/mm3, platelets ≥75,000/mm3 transfusion independent
  • Adequate renal, liver, and neurologic function
  • Arm A: Evaluable disease per RAPNO criteria, able to swallow capsules
  • Arm A: Non-fasting glucose ≤140 mg/dL, triglycerides and cholesterol <300 mg/dL, QTC <470 msec

Key Exclusion

  • Synchronous tumors or other extra-CNS malignancy
  • Currently receiving other investigational or anti-cancer agents
  • Uncontrolled infection or systemic illness
  • Pregnant or breast-feeding females
  • Arm A: Previous exposure to gemcitabine or paxalisib
  • Arm A: Concomitant use of antihyperglycemic agents
  • Arm A: Chronic diarrhea greater than Grade 2

NCT07226453

A Target Validation and Efficacy Study of Metformin in Patients With Recurrent or Progressive Posterior Fossa Group A (PFA) Ependymoma

Organization/Sponsor: University of California, San Francisco


Example patient: A 7-year-old child with recurrent posterior fossa A ependymoma after surgery and radiation, with measurable disease on MRI, normal blood counts and organ function, and no diabetes or active infections.

Phase 2

Interventions

  • Diagnostic Test: MR spectroscopy (MRS)
    Summary: Magnetic resonance spectroscopy is an imaging technique used to assess metabolic changes in tissues, source: NCI Thesaurus.
  • Procedure: Specimen Collection
    Summary: Obtaining biological samples like blood and tissue for medical testing to aid diagnosis and research in pediatric cancer trials, source: Summary of Web Search.
  • Diagnostic Test: Magnetic Resonance Imaging (MRI)
    Summary: Diagnostic imaging using magnetic fields and radio waves to create detailed images for detecting and monitoring brain cancer and assessing treatment effectiveness, source: Summary of Web Search and NCI Thesaurus.
  • Procedure: Planned Surgical Resection
    Summary: Surgical removal of tumor tissue chosen as optimal treatment considering all therapeutic options, source: NCI Thesaurus.
  • Drug: Metformin
    Summary: Metformin targets AMPK signaling to exert anti-cancer effects and is being studied for safety and efficacy in pediatric cancer, source: Summary of Web Search.

Key Inclusion

  • Recurrent or progressive posterior fossa A (PFA) ependymoma following surgery and radiation
  • Age 1-39 years at enrollment
  • Karnofsky ≥50 for >16 years or Lansky ≥50 for ≤16 years
  • Measurable disease with lesions ≥10 mm in two dimensions (Efficacy Phase)
  • Adequate tumor tissue available from initial diagnosis or pre-trial enrollment
  • ANC ≥1000/mm³ and platelet count ≥100,000/mm³
  • Serum creatinine <1.5 ULN, total bilirubin ≤1.5x ULN, ALT and AST ≤3x ULN
  • No prior metformin use for other indications or following PFA ependymoma diagnosis

Key Exclusion

  • History of diabetes mellitus or meeting pre-diabetes criteria
  • No measurable disease
  • Chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas)
  • Receiving other investigational agents
  • Concurrent radiation therapy
  • Pregnant or breast-feeding women
  • Uncontrolled intercurrent illness or active infection
  • Allergic reactions to metformin or similar compounds

NCT06465199

A Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas

Organization/Sponsor: Milton S. Hershey Medical Center


Example patient: A 9-year-old with relapsed high-risk neuroblastoma after completing induction chemotherapy and immunotherapy, now with stable bone metastases on recent salvage therapy, performance score of 70, and normal cardiac and renal function.

Phase 1, Phase 2

Interventions

  • Drug: AMXT 1501 Dicaprate
    Summary: An orally bioavailable polyamine transport inhibitor that blocks polyamine uptake into the tumor microenvironment, inhibiting tumor cell proliferation and inducing apoptosis while potentially reversing polyamine-mediated immune suppression (NCI Thesaurus).
  • Drug: Eflornithine (DFMO)
    Summary: An irreversible inhibitor of ornithine decarboxylase that blocks polyamine biosynthesis, inhibiting tumor cell formation and proliferation and inducing apoptosis (NCI Thesaurus).

Key Inclusion

  • Maximum 26 years of age at diagnosis
  • Relapsed/refractory neuroblastoma, ETMR, ATRT, Ewing sarcoma, or osteosarcoma; or newly diagnosed DIPG
  • DIPG patients must be 30-60 days post standard radiation therapy
  • Lansky or Karnofsky Performance Scale score ≥60
  • ANC ≥750/μL unsupported
  • Normal cardiac troponin, BNP, QTcF ≤470 msec, and ejection fraction ≥50%
  • eGFR ≥70 mL/min/1.73 m²
  • Able to swallow capsules

Key Exclusion

  • BSA <0.25 m²
  • Currently receiving other investigational drugs or anticancer agents
  • Uncontrolled infection
  • Pregnant or breastfeeding
  • DIPG patients with metastatic disease or progression after initial radiation
  • Neuroblastoma patients with active disease in organs (lungs, liver, or brain)
  • Unable to comply with safety monitoring requirements

NCT07464951

Phase 1 Trial of Autologous CD123-Directed CAR T-Cells (CART123) as Monotherapy or in Combination With Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia

Organization/Sponsor: Children's Hospital of Philadelphia


Example patient: A 14-year-old with relapsed AML after second remission showing 2% blasts by flow cytometry, adequate organ function, Karnofsky score of 60, and an available matched sibling donor.

Phase 1

Interventions

  • Drug: Ruxolitinib (JAKAVI®)
    Summary: Ruxolitinib is a JAK1/2 tyrosine kinase inhibitor that blocks Janus kinase signaling pathways involved in abnormal cell proliferation and immune-mediated inflammation. It is FDA-approved for myelofibrosis, polycythemia vera, and graft-versus-host disease in patients 12 years and older. Sources: FDA label, NCI Thesaurus.
  • Biological: Anti-CD123 LV redirected T cells (CART123)
    Summary: CART123 consists of autologous T-lymphocytes genetically engineered to express a chimeric antigen receptor targeting CD123, a protein overexpressed on AML cells. The CAR includes 4-1BB and CD3-zeta signaling domains to enhance T-cell activation and induce selective lysis of CD123-expressing tumor cells. Source: NCI Thesaurus.

Key Inclusion

  • Age 0-29 years (Cohort A) or 1-29 years (Cohort B)
  • AML in second or greater relapse, post-transplant relapse, or chemotherapy-refractory disease
  • Flow cytometric confirmation of myeloid leukemia ≥0.1% or >5% bone marrow blasts
  • Identified stem cell donor with ability to proceed rapidly to transplant
  • Adequate organ function including creatinine, liver function, pulmonary reserve, and cardiac function
  • Lansky or Karnofsky performance score ≥50

Key Exclusion

  • Active hepatitis B, hepatitis C, or HIV infection
  • Active acute or chronic GVHD requiring systemic therapy
  • Concurrent systemic steroids or immunosuppression at cell infusion or collection
  • CNS disease progressive on therapy or CNS parenchymal lesions increasing CNS toxicity risk
  • Pregnant or nursing
  • Uncontrolled active infection

NCT07407582

Phase I/II Open Label Trial of Intravenous Sodium Thiosulfate (Pedmark®) as Otoprotectant in Adults Receiving Cisplatin Chemotherapy (STOP-CIS)

Organization/Sponsor: University of Arizona


Example patient: A 45-year-old female with newly diagnosed ovarian cancer, ECOG status 0, normal electrolytes, scheduled for standard cisplatin-based chemotherapy with 4-hour infusions totaling 300 mg/m2.

Phase 1, Phase 2

Interventions

  • Drug: Pedmark® STS
    Summary: Sodium thiosulfate injection acts as a protective agent to reduce cisplatin-induced ototoxicity (hearing loss) by neutralizing platinum compounds. FDA-approved for pediatric patients with localized solid tumors; must be given within 6 hours of cisplatin infusion to prevent irreversible hearing damage (FDA label).

Key Inclusion

  • At least 18 years of age
  • ECOG Performance Status 0-1
  • Histologically or cytologically confirmed treatment-naïve cancer
  • Scheduled to receive FDA-approved cisplatin-based regimen (at least 200 mg/m2 cumulative dose)
  • Untreated solid malignancy

Key Exclusion

  • Prior cisplatin exposure
  • Concurrent ototoxic medication unable to be discontinued
  • Planned radiation to head or neck within 3 months of cisplatin completion
  • History of severe hypersensitivity to sulfite or sodium thiosulfate
  • Baseline serum sodium >145 mmol/L or grade ≥3 electrolyte abnormality
  • Cisplatin infusion duration greater than 6 hours
  • Pregnancy or breastfeeding

NCT07254793

A Feasibility/Pilot First-in-human Study of Exercise-mobilized NK-enriched Donor Lymphocyte Infusions (DLI-X) to Prevent or Treat Leukemia Relapse After Allogeneic Hematopoietic Cell Transplantation

Organization/Sponsor: University of Arizona


Example patient: A 14-year-old male with relapsed acute lymphoblastic leukemia undergoing matched sibling donor HCT with a healthy 35-year-old sibling donor who completed fitness evaluation, no active GvHD, and normal organ function.

Phase N/A

Interventions

  • Biological: Standard Therapeutic DLI
    Summary: Standard donor lymphocyte infusion uses donor T-cells to treat leukemia or lymphoma relapse post-transplant by leveraging graft-versus-tumor effect, though it can cause graft-versus-host disease. Source: Summary of Web Search.
  • Biological: Exercise Mobilized Therapeutic DLI
    Summary: Exercise-mobilized therapeutic DLI enhances anti-tumor activity by mobilizing NK-cells and T-cells with higher cytotoxicity to fight leukemia and lymphoma post-transplantation. Source: Summary of Web Search.
  • Biological: Standard Prophylactic DLI
    Summary: Prophylactic donor lymphocyte infusion prevents leukemia or lymphoma relapse post-transplant by stimulating graft-versus-tumor effect through immune system activation against residual malignant cells in high-risk patients. Source: Summary of Web Search.
  • Biological: Exercise Mobilized Prophylactic DLI
    Summary: Exercise-mobilized prophylactic DLI enhances donor lymphocyte infusions with exercise to improve immune response against leukemia relapse post-HCT, targeting leukemia cells with improved outcomes. Source: Summary of Web Search.

Key Inclusion

  • Aged 0-65 years
  • Diagnosis of acute leukemia, MDS, CML or NHL
  • Undergoing myeloablative or reduced intensity matched sibling or haploidentical HCT
  • Locally available healthy matched or haploidentical related donor aged 12-50 years
  • Donor able to complete fitness evaluation for VO2max and peak cycling power

Key Exclusion

  • Acute grade III-IV aGvHD or moderate/severe chronic GvHD
  • Requiring immunosuppression therapy for GvHD treatment
  • AST/ALT greater than 5x ULN or bilirubin greater than 2x ULN
  • Creatinine greater than 2x ULN or GFR less than 40 ml/min/1.73m2
  • DLCO less than 40% or O2 Sat less than 92%
  • Left ventricular ejection fraction less than 35%
  • Uncontrolled or severe bacterial, fungal or viral infection
  • Positive pregnancy test for females of childbearing age

NCT06786104

A Multisite RWCT Comparing the Effectiveness of a Goals of Care Video and Navigator Intervention, the VIDEO-PEDS Experience, Versus Usual Care in Pediatric Cancer

Organization/Sponsor: Massachusetts General Hospital


Example patient: A 7-year-old child with newly diagnosed acute lymphoblastic leukemia receiving chemotherapy, whose English-speaking biological mother is the primary decision maker and has not yet been referred to palliative care.

Phase N/A

Interventions

  • Behavioral: Focus Group
    Summary: A small diverse group used to study responses and determine expected outcomes from larger populations, applied in market research and analysis (NCI Thesaurus).
  • Behavioral: VIDEO-PEDS pilot
    Summary: Pilot intervention to improve communication between doctors and parents about pediatric cancer care goals using shared decision-making tools (Summary of Web Search).
  • Behavioral: VIDEO-PEDS
    Summary: Clinical intervention using video and navigator tools to enhance communication between doctors and parents regarding goals of care in pediatric cancer treatment (Summary of Web Search).

Key Inclusion

  • Age 0-12 years
  • Diagnosed with any type or stage of cancer
  • Receiving cancer directed treatment
  • Parent is decision maker for the child
  • Parent able to communicate in English or Spanish

Key Exclusion

  • Not receiving primary medical care from the cancer clinic
  • Already fully consulted by palliative care team
  • Prognosis of less than 2-month life expectancy
  • Parent visually impaired beyond 20/200 corrected and unable to view video
  • Parent psychological state not appropriate for goals of care discussions

NCT07306832

A Phase 1b Study of the Safety and Pharmacokinetics of Pivekimab Sunirine in Pediatric Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Organization/Sponsor: AbbVie


Example patient: A 12-year-old with CD123-positive acute myeloid leukemia in third relapse, 15% bone marrow blasts, Lansky score 60, no cardiac disease, no prior transplant or CAR-T therapy, and asymptomatic CNS2 status.

Phase 1

Interventions

  • Drug: Pivekimab Sunirine
    Summary: Antibody-drug conjugate targeting CD123 with cytotoxic payload, FDA approved for blastic plasmacytoid dendritic cell neoplasm in adults, studied for pediatric acute myeloid leukemia. Source: Web Search.

Key Inclusion

  • Histologically confirmed acute myeloid leukemia in second or greater relapse or refractory to second or subsequent line therapy
  • CD123-positive myeloid leukemic blasts by flow cytometry
  • ≥5% myeloid leukemic blasts in bone marrow
  • Lansky or Karnofsky performance status ≥50 or ECOG ≤2
  • CNS1, CNS2, or CNS3 disease without clinical neurologic symptoms allowed
  • May continue intrathecal therapy per institutional practice

Key Exclusion

  • Known clinically significant cardiac disease
  • Down syndrome
  • Acute promyelocytic leukemia or juvenile myelomonocytic leukemia
  • Symptomatic CNS3 disease
  • Prior veno-occlusive disease or sinusoidal obstructive syndrome of any severity
  • Hematopoietic stem cell transplant within 6 months or active GvHD
  • Prior CAR-T therapy
  • Currently receiving anticancer therapy except intrathecal treatment

NCT06914479

A Phase 1 Study of a Combined Cytotoxic and Immune-Stimulatory Therapy in Pediatric and Young Adult Patients With Recurrent, Primary Malignant Brain Tumors Genomic-based

Organization/Sponsor: University of Michigan Rogel Cancer Center


Example patient: A 12-year-old child with recurrent medulloblastoma who completed standard radiation and chemotherapy six weeks ago, weighs 35 kg, has stable neurological function with Lansky score of 80, and requires surgical resection of a new enhancing cerebellar mass.

Phase 1

Interventions

  • Procedure: Magnetic Resonance Imaging
    Summary: Noninvasive imaging using radiofrequency waves and magnetic fields to provide detailed pictures of internal organs and tissues, valuable for diagnosing cancer and monitoring treatment response (NCI Thesaurus).
  • Drug: Valacyclovir
    Summary: Nucleoside analog DNA polymerase inhibitor that converts to acyclovir, inhibiting viral DNA replication; used with HSV-tk gene therapy to kill tumor cells expressing thymidine kinase (FDA label, NCI Thesaurus).
  • Procedure: Tumor Resection
    Summary: Surgical procedure to remove cancerous tissue, often combined with other therapies to prevent recurrence and improve outcomes in pediatric cancer (NCI Thesaurus).
  • Other: Survey Administration
    Summary: Presentation of questionnaires to obtain patient responses, assessing experiences and knowledge through self-reported data collection (NCI Thesaurus).
  • Other: Biospecimen Collection
    Summary: Gathering tissue and fluid samples for testing and research to analyze genetic and molecular features, aiding in understanding cancer mechanisms and developing targeted therapies (NCI Thesaurus).
  • Biological: Ad-hCMV-TK
    Summary: Replication-defective adenoviral vector expressing HSV thymidine kinase under CMV promoter; converts prodrugs like valacyclovir into toxic compounds killing tumor cells and stimulating antitumor immune response (NCI Thesaurus).
  • Biological: Ad-hCMV-Flt3L
    Summary: Replication-defective adenoviral vector expressing Flt3 ligand under CMV promoter; stimulates dendritic cell proliferation and migration to tumor sites, initiating immune response against tumor-associated antigens (NCI Thesaurus).

Key Inclusion

  • Age 3 to 25 years with malignant primary brain tumor after recurrence, relapse, or progression
  • Age 26 to 39 years with diffuse hemispheric glioma, H3 G34-mutant after recurrence
  • Completed up-front, standard-of-care therapy
  • At least 10 kg and BSA > 0.5 m^2
  • Surgical resection of tumor recurrence clinically indicated
  • ANC ≥ 1000/mm^3 and platelet count ≥ 100,000/mm^3
  • Performance score ≥ 60 (Karnofsky or Lansky)
  • Recovered from all acute side effects of prior therapy with appropriate washout periods

Key Exclusion

  • Not clinically appropriate for tumor tissue resection
  • Evidence of disseminated disease or leptomeningeal disease
  • Primary brainstem or primary spinal tumors
  • History of prior gene therapy
  • Known allergy to valacyclovir or ongoing valacyclovir therapy unable to be stopped
  • Currently receiving other investigational or anti-cancer agents
  • Known immune disorder (HIV, hepatitis B/C, autoimmune disease requiring immunosuppressive therapy)
  • Pregnant or breast-feeding

NCT07637630

Applying the Multiphase Optimization Strategy to Develop and Test a Culturally-Relevant Digital Health Intervention Targeting HPV Vaccination Among U.S. Vietnamese

Organization/Sponsor: Northwestern University


Example patient: A 14-year-old Vietnamese-American adolescent with newly diagnosed leukemia whose parent is fluent in English, has lived in the U.S. for three years, and whose child has not received any HPV vaccine doses.

Phase N/A

Interventions

  • Behavioral: Constant Component
    Summary: An intervention component used in Master Trial approaches in pediatric cancer trials targeting specific tumor types to identify effective treatments with minimal exposure to ineffective therapies; mechanism not publicly disclosed (Summary of Web Search).
  • Behavioral: Motivational Interviewing
    Summary: A counseling method that enhances motivation for behavior change, shown effective in improving mental health treatment initiation and physical activity levels in pediatric cancer patients (Summary of Web Search).
  • Behavioral: Narrative Storytelling
    Summary: A technique used in pediatric oncology where children create stories to express experiences, targeting stress and anxiety to improve emotional well-being and symptom management (Summary of Web Search).
  • Behavioral: Self-persuasion
    Summary: A strategy to influence one's own beliefs and behaviors, targeting motivation and behavior change to improve treatment adherence and self-management in pediatric cancer patients (Summary of Web Search).
  • Behavioral: Expert Video
    Summary: A visual aid targeting adolescents to enhance understanding and engagement in pediatric oncology clinical trials, currently under development and evaluation (Summary of Web Search).

Key Inclusion

  • Self-identify as U.S. Vietnamese
  • Parent of youth ages 9-18
  • Youth have not initiated HPV vaccine series
  • One parent per household
  • Lived in U.S. for 12 months or more

Key Exclusion

  • Planning to move out of U.S. within next 6 months
  • Not fluent in spoken and written Vietnamese or English
  • Inability to provide informed consent