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Rady Children's Specialists

Active Clinical Trials

The Peckham Center for Cancer & Blood Disorders offers clinical trials on promising new treatments for children newly diagnosed with cancer or blood disorders or with recurrent tumors. We also offer studies investigating cancer biology, supportive care and late effects of treatment for children, adolescents and young adults.

Clinical trials for newly diagnosed patients or those with recurrent tumors are offered through our participation in the Children’s Oncology Group, our affiliation with St. Jude’s Children’s Research Hospital, and our participation in the Neuroblastoma and Medulloblastoma Translational Research Consortium. We also offer studies sponsored by pharmaceutical and biotechnology companies, as well as registry (observational) studies for patients with certain blood disorders, such as sickle cell anemia, hemophilia and thalassemia.

Before enrolling your child in a research study, you may want to talk to his or her physician about potential benefits and possible risks.

For more information about clinical trials, read these frequently asked questions.

For more information about the studies we offer and the rights of patients participating in clinical trials, please call Mehrzad Milburn at 858-966-8155 or email

Open Clinical Trials

Solid/CNS Tumors

Pilot: A Pilot Study Evaluating the Use of the mTOR Inhibitor Sirolimus in Children and Young Adults with Desmoid-Type Fibromatosis

Pilot: NF1 OPG: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1-Associated Optic Pathway Glioma 

Pilot: PNOC 007: H3.3K27M-Specific Peptide Vaccine Combined with Poly-ICLC for the Treatment of Newly Diagnosed HLA-A2+ H3.3K27M Positive Diffuse Intrinsic Pontine Glioma (DIPG) as well as Other Newly Diagnosed HLA-A2+ H3.3K27M Positive Gliomas

Phase I: A Phase 1/1b, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Children and Adolescents with Recurrent Solid Tumor and Primary CNS Tumors, with or without TPK, ROS1 or ALK Fusions

Phase I: A National Phase 1 Study of Cabozantinib in Combination with 13-cis-Retinoic Acid in Children with Relapsed or Refractory Solid Tumors, including CNS Tumors

Phase I: PNOC 013: A Safety and Pharmacokinetic Study of Single Agent REGN2180 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 

Phase II: NMTRC012: PEDS-PLAN – Pediatric Precision Laboratory Advanced Neuroblastoma Therapy. A Study Using Molecular-Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of Standard Immunotherapy with or without DFMO Followed by DFMO Maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma

Phase II: NMTRC014: Neuroblastoma Maintenance Therapy Trial Using Difluromethylornithine (DFMO)

Phase II: SJATRT: A Phase 2 Study of Alisertib as a Single Agent in Recurrent or Progressive Central Nervous System (CNS) Atypical Teratoid Rhabdoid Tumors (ATRTs) and Extra-CNS Malignant Rhabdoid Tumors (MRTs) and in Combination Therapy in Newly Diagnosed ATRT (St. Jude)

Phase II: PNOC 001: A Phase 2 Study of Everolimus for Recurrent or Progressive Low-Grade Gliomas in Children

Phase II: PNOC 010: A Phase 2 Study of Binimetinib in Children and Adults with NF1-associated Plexiform Neurofibromas

Phase II: COG ADVL1722: A Phase 2, Multicenter, Open-Label Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Subjects with Relapsed/Refractory Rhabdomyosarcoma (RMS), Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS) and Ewing Sarcoma (EWS)

Phase III: SJMB12: A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma (St. Jude)

Phase III: LGG 14C03: A Phase 3 Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults with Previously Untreated Low-Grade Glioma


Phase I/II: TINI: Total Therapy for Infants with Acute Lymphoblastic Leukemia (ALL) (St. Jude)

Phase II: COG AALL1521: A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib with Chemotherapy in Children with De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia

Phase II: COG AALL1721: A Phase 2 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

Phase II: AML16: A Phase 2 Trial of Epigenetic Priming in Patients with Newly Diagnosed Acute Myeloid Leukemia (St. Jude)

Phase III: SJBC3: Mature B-Cell Lymphoma and Leukemia Study III (St. Jude)

Phase III: ACCL1333: A Phase 3 Randomized, Open-Label, Multi-center Study of the Safety and Efficacy of Apixaban for Thromboembolism Prevention Versus No Systemic Anticoagulant Prophylaxis during Induction Chemotherapy in Children with Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) or Lymphoma (T or B cell) Treated with Asparaginase

Phase III: Randomization of Cytarabine Monotherapy versus Standard-of-care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCG LCH0115)

Cell Therapy/Bone Marrow Transplantation

Pilot: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime with Campath-1H, Fludarabine and Melphalan

Phase I/II: PCYC-1146-IM: Phase I/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Phase II: PBMTC ONC1401: The Role of KIR-favorably Mismatched Haploidentical Transplantation and KIR-polymorphisms in Determining Outcomes of Children with ALL/AML/MDS Undergoing Allogeneic Hematopoietic Cell Transplantation


kids-DOTT Prospective Evaluation of the Duration of Therapy for Thrombosis in Children

Phase III: ALN-AT3SC-003: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

Phase III: ALN-AT3SC-004: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, without Inhibitory Antibodies to Factor VIII or IX

Phase III: INdividualized ITI Based on fVIII(ATE) Protection by VWF (INITIATE)

Phase III: A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs. Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant