Know Your Options

Not sure which clinical trials might be right for your child? Email our clinical trials team at clinicaltrials@danafarberbostonchildrens.org.
We can help you navigate your options.

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Get answers to frequently asked questions about clinical trials: What are clinical trials? Is a clinical trial the right choice for my child?

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Clinical Trials for Relapsed Cancer

For patients with relapsed, refractory, or recurrent cancer, early phase clinical trials may be a treatment option. Phase I and phase II clinical trials provide access to the newest, most innovative therapies to patients.

The Advancing Childhood Cancer Therapies (ACCT) Clinic is dedicated to offering state-of-the-art therapies, advancing the science of pediatric research, and providing personalized care to you and your family. The Clinic includes experts in each type of cancer as well as teams of subspecialists and nurses who are leaders in their respective fields. In August 2016, we joined the Children’s Oncology Group Phase 1 Consortium, one of only 21 such centers in the United States and the only one in New England.

Our currently active phase I and phase II trials for patients with relapsed, recurrent, or refractory cancer are listed below by disease.

If you have questions about which trial might be right for your child or patient, please email us at clinicaltrials@danafarberbostonchildrens.org or call us at 1-855-320-2090. We can help you navigate your options.

Phase I and II Trials for Relapsed and Refractory Cancer

    Brain Tumor Trials
    1. A Phase I of DAY101 (formerly TAK-580, MLN2480) for Children with Low-Grade Gliomas and Other RAS/RAF/MEK/ERK Pathway Activated Tumors
      Principal Investigator Karen Wright, MD
      Status: Active/Recruiting
    2. A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
      Principal Investigator Susan Chi, MD
      Status: Active/Recruiting
    3. Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer
      Principal Investigator David Shulman, MD
      Status: Active/Recruiting
    4. A Phase I Study of Selinexor (KPT-330, IND 125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    5. A Phase I/II Study of MK-3475 (Pembrolizumab) in Children with advanced melanoma or a PD-L1 positive advanced, relapsed or refractory solid tumor or lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    6. Phase 1 Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    7. A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    8. Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    9. A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK-3 Alterations
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    10. Phase 2 Proof of Concept Study of Nivolumab and Ipilimumab in Children and Young Adults with Relapsed or Refractory INI1-Negative Cancers
      Principal Investigator Suzanne Forrest, MD
      Status: Active/Recruiting
    11. An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
      Principal Investigator Sarah Nikiforow, MD, PhD
      Status: Active/Recruiting
    12. PNOC 017: A Target Validation/Phase1 Study of BGB-290 in Combination with Temozolomide in Adolescent and Young Adult IDH1/2 Newly Diagnosed and Recurrent Mutant Gliomas
      Principal Investigator Susan Chi, MD
      Status: Active/Recruiting
    13. A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)
      Principal Investigator Susan Chi, MD
      Status: Active/Recruiting
    14. A Phase I Study of the Adam-10 Inhibitor, INCB7839 in Children with Recurrent/Progressive High-Grade Gliomas to Target Microenvironmental Neuroligin-3
      Principal Investigator Katherine Warren, MD
      Status: Active/Recruiting
    15. NF110: Open-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults with Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas
      Principal Investigator Nicole Ullrich, MD, PhD
      Status: Active/Recruiting
    16. FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients with BRAF-Altered, Recurrent or Progressive Low-Grade Glioma
      Principal Investigator Susan Chi, MD
      Status: Active/Recruiting
    17. A Phase II Study of Binimetinib in Children and Adults with NF1 associated Plexiform Neurofibromas (PNOC010)
      Principal Investigator Nicole Ullrich, MD, PhD
      Status: Active/Not Recruiting
    18. A Phase II Study of Metronomic and Targeted Anti-Angiogenesis Therapy for Children with Recurrent/Progressive Medulloblastoma
      Principal Investigator Susan Chi, MD
      Status: Active/Not Recruiting
    19. Phase I/II Study of MEK162 for Children Low-Grade Gliomas and Other Ras/Raf/ERK Pathway Activated Tumors
      Principal Investigator Karen Wright, MD, MS
      Status: Active/Not Recruiting
    20. PNOC 001:Phase II Study of Everolimus for Recurrent or Progressive Low-Grade Glioma in Children
      Principal Investigator Susan Chi, MD
      Status: Active/Not Recruiting
    21. A Phase II, Multicenter Study of the EZH2 Inhibitor Tazemetostat in Adult Subjects with INI1-Negative Tumors or Relapsed/Refractory Synovial Sarcoma
      Principal Investigator Gregory Cote, MD
      Status: Active/Not Recruiting
    22. A Phase 1/2 Study of AZD1775 (MK-1775, IND# 121422) in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    23. Bayer 20810 - A Phase 1/2 Study of the TRK Inhibitor BAY 2731954 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers. Formerly: LOXO-EXT-17005 - A Phase 1/2 Study of the TRK Inhibitor LOXO-195 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancer
      Principal Investigator Jessica Lin, MD
      Status: Active/Not Recruiting
    24. Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Not Recruiting
    25. A Phase 1 Study of ABI-009 (nab-rapamycin) (IND# 133537) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    26. A Phase 1/2 Study of VX15/2503 (IND# 136181), Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    Leukemia Trials
    1. Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    2. Phase I Trial of Ribociclib in Combination with Everolimus and Dexamethasone in Children and Young Adults with Relapsed Acute Lymphoblastic Leukemia
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    3. A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia
      Principal Investigator Yana Pikman, MD
      Status: Active/Recruiting
    4. A Phase II Trial of Tisagenlecleucel in First-Line High-Risk (HR) Pediatric and Young Adult Patients with B-Cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    5. A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients with Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
      Principal Investigator Richard Stone, MD
      Status: Active/Recruiting
    6. ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    7. Phase 1/2 Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults with Relapsed/Refractory Acute Myeloid Leukemia
      Principal Investigator Susanne Baumeister, MD
      Status: Active/Recruiting
    8. A Phase I/II Study of Nivolumab in Combination with 5-azacytidine in pediatric patients with relapsed/refractory acute myeloid leukemia
      Principal Investigator Jessica Pollard, MD
      Status: Active/Recruiting
    9. A Phase I/II Study of Bosutinib in Pediatric Patients With Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph+ Chronic Myeloid Leukemia, Study ITCC-054/ COG AAML 1921
      Principal Investigator Jessica Pollard, MD
      Status: Active/Recruiting
    Lymphoma Trials
    1. A Phase I Study of Selinexor (KPT-330, IND 125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    2. A Phase I/II Study of MK-3475 (Pembrolizumab) in Children with Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    3. Phase 1 Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    4. A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    5. A Non-Randomized, Open-Label, Multi-Center, Phase I/II Study of PI3K Inhibitor Copanlisib in Pediatric Patients with Relapsed/Refractory Solid Tumors or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    6. Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    7. A Phase II, Single Arm, Multicenter Open Label Trial to Determine the Safety and Efficacy of Tisagenlecleucel in Pediatric Patients with Relapsed or Refractory Mature B-Cell Non-Hodgkin Lymphoma (NHL) (BIANCA)
      Principal Investigator Kimberly Davies, MD
      Status: Active/Recruiting
    8. Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    9. Phase I/II, Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Durvalumab Monotherapy or Durvalumab in Combination with Tremelimumab in Pediatric Patients with Advanced Solid Tumors and Hematological Malignancies
      Principal Investigator Natalie Collins, MD, PhD
      Status: Active/Recruiting
    10. A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK-3 Alterations
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    11. Pediatric Classical Hodgkin Lymphoma Consortium Study: cHOD17
      Principal Investigator Angela Feraco, MD, MMSc
      Status: Active/Recruiting
    12. An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
      Principal Investigator Sarah Nikiforow, MD, PhD
      Status: Active/Recruiting
    13. Phase I study of carfilzomib in combination with cyclophosphamide and etoposide for children with relapsed and refractory solid tumors and leukemias
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    14. A Phase 1/2 Study of Nivolumab (IND# 124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    15. A Phase 1/2 Study of VX15/2503 (IND# 136181), Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    Neuroblastoma Trials
    1. A Phase I Study of Selinexor (KPT-330, IND 125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    2. Phase 1 Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    3. Phase 1 Study of Lorlatinib (PF-06463922), an Oral Small Molecule Inhibitor of ALK/ROS1, for Patients with ALK-Driven Relapsed or Refractory Neuroblastoma
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    4. A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    5. NANT 2017-01: A Phase I Study Of 131I-MIBG With Dinutuximab For Relapsed/Refractory Neuroblastoma
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    6. A Phase II, Open Label Two Arm Study of Therapeutic Iobenguane (131I) as Single Agent or in Combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (Optimum Trial)
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    7. Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    8. A Phase 1 Study of Aurora Kinase A Inhibitor LY3295668 Erbumine as a Single Agent and in Combination in Patients with Relapsed/Refractory Neuroblastoma
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    9. A Phase 1 Study of Combination Nivolumab and Ipilimumab with Irradiated GM-CSF Secreting Autologous Neuroblastoma Cell Vaccine (GVAX) for Relapsed or Refractory Neuroblastoma
      Principal Investigator Natalie Collins, MD PhD
      Status: Active/Recruiting
    10. A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia
      Principal Investigator Yana Pikman, MD
      Status: Active/Recruiting
    11. Phase I Study of Carfilzomib in Combination with Cyclophosphamide and Etoposide for Children with Relapsed and Refractory Solid Tumors and Leukemias
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    12. A Phase 1/2 Study of AZD1775 (MK-1775, IND# 121422) in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    13. A Phase 1/2 Study of Nivolumab (IND# 124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    14. Bayer 20810 - A Phase 1/2 Study of the TRK Inhibitor BAY 2731954 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers. Formerly: LOXO-EXT-17005 - A Phase 1/2 Study of the TRK Inhibitor LOXO-195 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancer
      Principal Investigator Jessica Lin, MD
      Status: Active/Not Recruiting
    15. A Phase 1 Study of ABI-009 (nab-rapamycin) (IND# 133537) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    16. A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Not Recruiting
    Kidney, Liver, and Other Solid Tumor Trials
    1. Phase I Study of Olaparib with Irinotecan and/or Temozolomide in Adolescent and Adult Patients with Recurrent/Metastatic Ewing Sarcoma Following Failure of Prior Chemotherapy
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    2. A Phase I Study of Selinexor (KPT-330, IND 125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    3. A Phase I/II Study of MK-3475 (Pembrolizumab) in Children with Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    4. Phase 1 Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    5. A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    6. A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
      Principal Investigator David Ebb, MD
      Status: Active/Recruiting
    7. Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    8. Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    9. Phase I/II, Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Durvalumab Monotherapy or Durvalumab in Combination with Tremelimumab in Pediatric Patients with Advanced Solid Tumors and Hematological Malignancies
      Principal Investigator Natalie Collins, MD, PhD
      Status: Active/Recruiting
    10. The Role of Checkpoint Inhibition in Relapsed/Refractory Pediatric Hepatocellular Carcinoma: Clinical Efficacy and Biologic Correlates. A Phase II Study.
      Principal Investigator Allison O'Neill, MD
      Status: Active/Recruiting
    11. A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK-3 Alterations
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    12. Phase 2 Proof of Concept Study of Nivolumab and Ipilimumab in Children and Young Adults with Relapsed or Refractory INI1-Negative Cancers
      Principal Investigator Suzanne Forrest, MD
      Status: Active/Recruiting
    13. ARST1921 A Safety, Pharmacokinetic and Efficacy Study of a Secretase Inhibitor, Nirogacestat (PF-03084014; IND# 146375, in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors A Groupwide Phase 2 Study
      Principal Investigator Katherine Janeway, MD, MMSc
      Status: Active/Recruiting
    14. An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
      Principal Investigator Sarah Nikiforow, MD, PhD
      Status: Active/Recruiting
    15. An Open-Label, Dose Escalation, Multi-Center Phase I/II Clinical Trial of ET140203 T Cells in Pediatric Subjects with Relapsed/Refractory Hepatoblastoma (HB), Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS), or Hepatocellular Carcinoma (HCC)
      Principal Investigator Allison O'Neill, MD
      Status: Active/Recruiting
    16. Phase 1 Study of the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Activity of RP-6306 in Patients with Advanced Solid Tumors (MYTHIC Study)
      Principal Investigator Elizabeth Lee, MD
      Status: Active/Recruiting
    17. Phase I Study of Carfilzomib in Combination with Cyclophosphamide and Etoposide for Children with Relapsed and Refractory Solid Tumors and Leukemias
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    18. A Phase 1/2, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Pediatrics and Young Adults with no Curative First-Line Treatment Option or Recurrent/Refractory Solid Tumors and Primary CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Not Recruiting
    19. A Phase 1/2 Study of AZD1775 (MK-1775, IND# 121422) in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    20. A Phase 1/2 Study of Nivolumab (IND# 124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab
      Principal Investigator Steven DuBois, MD, MS
    21. A Phase 1/2 Study of Nivolumab (IND# 124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    22. Bayer 20810 - A Phase 1/2 Study of the TRK Inhibitor BAY 2731954 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers. Formerly: LOXO-EXT-17005 - A Phase 1/2 Study of the TRK Inhibitor LOXO-195 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancer
      Principal Investigator Jessica Lin, MD
      Status: Active/Not Recruiting
    23. Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Not Recruiting
    24. A Phase 1 Study of ABI-009 (nab-rapamycin) (IND# 133537) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    25. A Phase 1/2 Study of VX15/2503 (IND# 136181), Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    Bone Tumor and Other Sarcoma Trials
    1. Phase I Study of Olaparib with Irinotecan and/or Temozolomide in Adolescent and Adult Patients with Recurrent/Metastatic Ewing Sarcoma following failure of prior chemotherapy
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    2. A Phase I Study of Selinexor (KPT-330, IND 125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Recruiting
    3. A Phase I/II Study of MK-3475 (Pembrolizumab) in Children with Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    4. Phase 1 Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    5. Phase 1 Trial of the LSD1 Inhibitor SP-2577 in Patients with Relapsed or Refractory Ewing Sarcoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    6. A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies
      Principal Investigator Andrew Place, MD, PhD
      Status: Active/Recruiting
    7. A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
      Principal Investigator David Ebb, MD
      Status: Active/Recruiting
    8. Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    9. A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    10. Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    11. Phase I/II, Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Durvalumab Monotherapy or Durvalumab in Combination with Tremelimumab in Pediatric Patients with Advanced Solid Tumors and Hematological Malignancies
      Principal Investigator Natalie Collins, MD, PhD
      Status: Active/Recruiting
    12. Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    13. A Phase 2 Trial of the MEK inhibitor selumetinib (AZD6244 hydrogen sulfate) in combination with the mTOR inhibitor sirolimus for patients with unresectable or metastatic malignant peripheral nerve sheath tumors
      Principal Investigator Natalie Collins, MD, PhD
      Status: Active/Recruiting
    14. A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK-3 Alterations
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    15. ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    16. Phase 2 Proof of Concept Study of Nivolumab and Ipilimumab in Children and Young Adults with Relapsed or Refractory INI1-Negative Cancers
      Principal Investigator Suzanne Forrest, MD
      Status: Active/Recruiting
    17. Phase II Trial of Olaparib in Combination with Ceralasertib in Patients with Recurrent Osteosarcoma
      Principal Investigator Katherine Janeway, MD, MMSc
      Status: Active/Recruiting
    18. Phase 1 Study of Cabozantinib in Combination with Topotecan-Cyclophosphamide for Patients with Relapsed Ewing Sarcoma or Osteosarcoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Recruiting
    19. ARST1921 A Safety, Pharmacokinetic and Efficacy Study of a Secretase Inhibitor, Nirogacestat (PF-03084014; IND# 146375, in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors A Groupwide Phase 2 Study
      Principal Investigator Katherine Janeway, MD, MMSc
      Status: Active/Recruiting
    20. An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
      Principal Investigator Sarah Nikiforow, MD, PhD
      Status: Active/Recruiting
    21. A Phase II, Multicenter Study of the EZH2 Inhibitor Tazemetostat in Adult Subjects with INI1-Negative Tumors or Relapsed/Refractory Synovial Sarcoma
      Principal Investigator George Demetri, MD
      Status: Active/Not Recruiting
    22. A Phase 1/2 Study of AZD1775 (MK-1775, IND# 121422) in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    23. A Phase 1/2 Study of Nivolumab (IND# 124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    24. Phase 1 Study of Pazopanib in Combination with Irinotecan and Temozolomide (PAZIT) for Children and Young Adults with Relapsed or Refractory Sarcoma
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    25. Bayer 20810 - A Phase 1/2 Study of the TRK Inhibitor BAY 2731954 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers. Formerly: LOXO-EXT-17005 - A Phase 1/2 Study of the TRK Inhibitor LOXO-195 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancer
      Principal Investigator Jessica Lin, MD
      Status: Active/Not Recruiting
    26. Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
      Principal Investigator Suzanne Shusterman, MD
      Status: Active/Not Recruiting
    27. A Phase 1 Study of ABI-009 (nab-rapamycin) (IND# 133537) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    28. A Phase 1/2 Study of VX15/2503 (IND# 136181), Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid Tumors
      Principal Investigator Steven DuBois, MD, MS
      Status: Active/Not Recruiting
    Stem Cell Transplant and Cellular Therapies
    1. Acute GVHD Supression Using Costimulation Blockade to Expand Non-Malignant Transplant (ASCENT)
      Principal Investigator John Horan, MD, MPH
      Status: Active/Recruiting