Clinical Research Trials
CURRENT TRIALS
CLINICAL TRIAL/CANCER TYPE
SUMMARY DESCRIPTION
TRIAL INFORMATION
Blood-Based Biomarkers for Minimal Residual Disease Detection in Pediatric Sarcomas.
This study is trying to see if testing blood for pieces of DNA (ctDNA) and cancer cells from the tumor (CTCs) floating around in the blood can be used to predict if cancer will come back in patients who seem to be in remission (no signs of cancer).
MCC 20320
M. Hayashi, MD
Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma.
This clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic Fusion Positive (alveolar) Rhabdomyosarcoma. The participant and their physician will choose from: Arm A) a first strike therapy, Arm B) a first strike-second strike (maintenance) therapy, Arm C) an adaptively timed therapy, and Arm D) conventional chemotherapy.
Interventional Phase II
NCT04388839
J. Metts, MD
A Multi-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Osteosarcoma: Bone and Soft Tissue.
The purpose of this study is to determine if the addition of infusions of a type of immune cell called a “natural killer”, or NK cell to the sarcoma chemotherapy regimen GEM/DOX (gemcitabine and docetaxel) can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies.
Interventional Phase I and II
NCT05634369
B. Setty, MD
Generating Novel Translational and Therapeutic Strategies Based on a Multicenter, Pediatric and AYA Evolutionary Tumor Board (pedsETB) to develop additional therapeutic strategies in patients without curative options or with suboptimal outcomes.
The pedsETB consists of evolutionary biologists, mathematicians, research scientists, statisticians, data scientists, radiologists, pathologists, oncologists (surgical, radiation, medical, and pediatric), and clinical trial coordinators. The pedsETB membership is inclusive of members from the participating institutions. The pedsETB will generate hypotheses, mathematical models, and experiments from the discussion towards further integration of evolutionary ideas towards therapeutic strategies for participants. The pedsETB will collect data through a chart review regarding adherence and results of ETB recommendation.
Interventional Phase II
NCT06423950
J. Metts, MD
Evaluation of Digoxin for Relapsed Non-WNT, Non-SHH Medulloblastoma.
The purpose of this study is to evaluate the efficacy of digoxin in treating relapsed non-SHH, non-WNT medulloblastoma in pediatric and young adult patients.
Interventional Phase II
NCT06701812
M. Isakoff, MD & L. Metrock, MD
A Proposed Trial Design for the Treatment of Widely Metastatic Ewing Sarcoma Inspired by Evolutionary Dynamics.
Metastatic Ewing sarcoma has dismal long-term survival despite multiple attempts to intensify standard therapy through the addition of new agents to the existing chemotherapy backbone. Here, based on the application of evolutionary dynamics to pediatric sarcoma, we propose an alternative treatment strategy that varies exposure to agents and dosing intensities, termed sequential second-strike therapy (SSST). We announce an upcoming clinical trial to apply these principles to patients with widely metastatic Ewing sarcoma, those with metastatic disease beyond the lungs.
MCC 23281
J. Metts, MD & M. Trucco, MD
RNA PRIME – RNA Lipid Particles Targeting Pediatric Recurrent Intracranial Malignancies and Other systEmic Solid Tumors.
This study tests the safety, immune effects, and potential effectiveness of RNA-loaded lipid nanoparticles (RNA-LP) vaccines in patients with recurrent pediatric High-Grade Glioma (pHGG), a type of brain cancer, and recurrent Osteosarcoma (OSA), a type of bone cancer. Based on earlier lab research and experience with similar vaccines for kids with brain tumors, we believe this treatment could help trigger immune responses that fight the cancer. This approach may offer benefits for patients with these cancers, where other treatments haven’t been able to cure the disease.
NCT05660408
J. Ligon, MD & E. Hwang, MD
Vuja De – Open-label, cohort-sequential dose-escalation and dose-confirmation Phase 1/2 clinical trial to evaluate the safety and efficacy of domatinostat in combination with sirolimus in adolescents and adults with relapsed, refractory sarcoma and osteosarcoma.
The Vuja De study is a clinical trial that tests the safety and effectiveness of two drugs, domatinostat and sirolimus, used together in patients with Osteosarcoma and other sarcomas that have come back or don’t respond to other treatments. This study will gradually increase the drug doses to find the best and safest amount for patients.
Clinical Trial TBD
A. Armstrong, MD
COMPLETED TRIALS
CLINICAL TRIAL/CANCER TYPE
SUMMARY DESCRIPTION
TRIAL INFORMATION
Phase II Study of nab-Paclitaxel in Combination with Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults.
The purpose of this study is to see if nab-paclitaxel combined with gemcitabine prevents the formation or growth of tumors in participants with relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and other soft tissue sarcoma and to measure the length of time during and after treatment that their disease does not get worse. Researchers also want to find out if nab-paclitaxel combined with gemcitabine is safe and tolerable.
NCT02945800
J. Oesterheld, MD
A Phase Ib/II Study to Evaluate the Safety, Feasibility, and Efficacy of Nivolumab in Combination with Azacitidine in Patients with Recurrent, Resectable Osteosarcoma.
The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab in combination with azacitidine in participants with recurrent, resectable osteosarcoma.
NCT03628209
P. Thompson, MD & M. Druta, MD
Phase I Trial of the LSD1 Inhibitor Seclidemstat (SP 2577) With and Without Topotecan and Cyclophosphamide in Patients With Relapsed or Refractory Ewing Sarcoma and Select Sarcomas.
Single agent, non-randomized, open label expansion in select sarcoma patients including myxoid liposarcoma and other sarcomas that share similar chromosomal translocations to Ewing sarcoma; AND dose expansion of the combination of seclidemstat with topotecan and cyclophosphamide in patients with Ewing sarcoma.
NCT03600649
D. Reed, MD
ACTION Trial: Adoptive Cellular Therapy Following Dose-Intensified Temozolomide in Newly-diagnosed Pediatric High-grade Gliomas (Phase I).
This trial is testing a type of immunotherapy to see if it is safe for children with High-Grade Gliomas (HGG), a type of brain cancer. It focuses on patients who have already received a stronger dose of the chemotherapy drug temozolomide and a special immune treatment using their own immune cells (Dendritic Cells and Autologous Lymphocyte Transfer). Some patients will also receive their own stem cells as part of the treatment. Researchers will also check if patients can complete the full treatment and track how long they stay cancer-free and how well they respond overall.
ACTION Trial
NCT03334305
D. Mitchell, MD, PhD
A Phase I Trial of Dose Escalation of Metformin in Combination With Vincristine, Irinotecan, and Temozolomide in Children With Relapsed or Refractory Solid Tumors.
Metformin is an oral anti-diabetes medication that activates AMP-activated protein kinase (AMPK). Recent data from in vitro and in vivo experiments, as well as epidemiologic retrospective analyses, suggest that metformin has anti-cancer activity. Vincristine, irinotecan, and temozolomide (VIT) is a combination of chemotherapeutic agents that have different mechanisms of action as well as disparate side effect profiles. Two recent phase 1 trials have demonstrated that this regimen is safe and well-tolerated in children with relapsed and refractory solid tumors.
NCT01528046
D. Reed, MD
A Phase I Window, Dose Escalating and Safety Trial of Metformin in Combination With Induction Chemotherapy in Relapsed Refractory Acute Lymphoblastic Leukemia: Metformin With Induction Chemotherapy of Vincristine, Dexamethasone, Doxorubicin, and PEG-asparaginase (VPLD).
This will be a phase I protocol of Vincristine, Dexamethasone, Doxorubicin, and PEG-asparaginase (VPLD) and metformin conducted in the Sunshine Project sites for children with recurrent ALL. All sites will be eligible to open this study, provided they agree to adhere to all study procedures and make a good faith effort to obtain all pharmacodynamic and pharmacokinetic evaluations requested.
NCT01324180
J. Goldberg, MD
Phase I, Traditional 3+3, Trial of PO Sorafenib and Topotecan in Refractory or Recurrent Pediatric Solid Malignancies.
The purpose of this research study is to establish a dose of the combination of drugs, Topotecan and Sorafenib in children. This will be called the maximum tolerated dose. The chemotherapy in this study is a combination of Topotecan and Sorafenib. The investigators are trying to find the highest dose of Topotecan and Sorafenib that can be given safely to children with Refractory or Recurrent Pediatric Solid Malignancies. The investigators will do this by testing different doses of these drugs in different groups of children. The investigators will also study how the body processes these drugs.
NCT01683149
D. Reed, MD
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