Phase I / Phase II Protocols / Clinical Studies

For information on these and other studies, contact the Developmental Therapeutics team at


A Phase II Study of Therapy for Pediatric Relapsed or Refractory Precursor B-Cell Acute Lymphoblastic Leukemia and Lymphoma

For: Relapsed or refractory precursor B-cell acute lymphoblastic leukemia and lymphoma


A Randomized Trial of Clofarabine Plus Cytarabine Versus Conventional Induction Therapy and of Natural Killer Cell Transplantation Versus Conventional Consolidation Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia

For: acute myeloid leukemia (AML)


A Phase I Study of Immunotherapy Including Haploidentical NK Cell Infusion Following CD133+Positively-Selected Autologous Hematopoietic Stem Cells In Children With High-Risk Solid Tumors or Lymphomas

For: high-risk solid tumors and lymphomas


A Phase I Trial of Bendamustine in Combination with Clofarabine and Etoposide in Pediatric Patients with Relapsed or Refractory Hematologic Malignancies

For: Relapsed or refractory hematologic malignancies


Therapeutic Trial for Newly Diagnosed Patients with Ewing Sarcoma Family of Tumor and Desmoplastic Small Round Cell Tumors

For: Ewing Sarcoma Family of Tumor and Desmoplastic Small Round Cell Tumors


Adcetris® (Brentuximab Vedotin) Substituting Vincristine in the OEPA/COPDac Regimen [Treatment Group 3 (TG3) of Euro-Net C1] With Involved Node Radiation Therapy for High Risk Pediatric Hodgkin Lymphoma (HL) Diseases: High risk Hodgkin Lymphoma

For: High risk Hodgkin lymphoma


Neuroblastoma Protocol 2012: Therapy for Children with Advanced Stage High-Risk Neuroblastoma

For: Neuroblastoma


Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Congestive Heart Failure (PREVENT-CHF):A Phase IIB Randomized Placebo-Controlled Trial

For: congestive heart failure


A Phase I Trial of RAD001 (Everolimus) and AVASTIN (Bevacizumab) in Children with Recurrent Solid Tumors

For: Refractory/relapsed solid tumor or brain tumor


A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children with Relapsed or Refractory Solid Tumors or Lymphomas

For: Relapsed or refractory solid tumors or lymphomas


A Pilot Pharmacokinetic, Pharmacodynamic, and Feasibility Study of Sorafenib in Combination with Cytarabine and Clofarabine in Patients with Refractory or Relapsed Hematologic Malignancies

For: Acute myeloid leukemia (AML), acute promyelocytic leukemia (APL), acute lymphoblastic leukemia (ALL), infantile leukemia (both AML and ALL), AML w/prior myelodysplastic syndrome (MDS), myelodysplastic / myeloproliferative neoplasms, biphenotypic leukemia


A Phase I Pharmacokinetic, Pharmacodynamic and Feasibility Study of Crenolanib in Combination with Sorafenib in Patients with Refractory or Relapsed Hematologic Malignancies (ARO-008)

For: Refractory or relapsed hematologic malignancies (leukemia)


A Phase II Trial of Surgery and fractionated Re-Irradiation for Recurrent Ependymoma

For: recurrent ependymoma


Risk Adapted Focal Proton Beam Radiation and/or Surgery in Participants with Low and Intermediate Risk Rhabdomyosarcoma Receiving Standard or Intensified Chemotherapy

For: Rhabdomyosarcoma (Low-risk and intermediate-risk)


A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma and Observation for Craniopharyngioma after Radical Resection

For: Craniopharyngioma


A Phase I/II Study of the Selective Inhibitor of Nuclear Export Selinexor (KPT-330) in Combination with Fludarabine and Cytarabine in Pediatric Patients with Refractory or Relapsed Leukemia or Myelodysplastic Syndrome

For: Relapsed or refractory leukemia or hematologic malignancies


A Phase I Study of Unmodified Live Intranasal Sendai Virus Vaccine in Children and Toddlers: Assessment of Safety and Immunogenicity

For: Croup


Phase II Study of Alisertib As A Single Agent in Recurrent and Refractory Central Nervous System Atypical Teratoid Rhabdoid Tumors (ATRT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnosed ATRT

For: Brain Tumor: Progressive or newly diagnosed Atypical Teratoid Rhabdoid Brain Tumors (ATRT). Solid Tumor: Progressive extra-CNS Malignant Rhabdoid Solid Tumors (MRT)


A Phase I trial of the humanized anti-GD2 antibody (hu14.18K322A) in children and adolescents with neuroblastoma, osteosarcoma and melanoma

For: Neuroblastoma, osteosarcoma and melanoma


Phase I Study of the Combination of Crizotinib and Dasatinib in Pediatric Research Participants with Diffuse Intrinsic Pontine Glioma and High-Grade Glioma

For: Progressive or recurrent pontine glioma or high-grade glioma


A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma

For: Medulloblastoma (includes all variants of medulloblastoma and posterior fossa PNET)


Protocol for the Study and Treatment of Participants with Intraocular Retinoblastoma

For: Retinoblastoma


Non St. Jude Protocols


A Phase I Study of Crizotinib (IND#105573) In Combination with Conventional Chemotherapy For Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma

For: Relapsed or refractory solid tumors or anaplastic large cell lymphoma


A Phase 1 Study of MK-1775 (IND#116495) Concurrent with Local Radiation Therapy For The Treatment Of Newly Diagnosed Children With Diffuse Intrinsic Pontine Gliomas (ADVL1217)

For: Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)


A Phase 1/2 Study of MK-1775 (AZD1775, IND# 121422) in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors (ADVL1312)

For: Relapsed or refractory solid tumors


A Phase I/II Study of PF-02341066, An Oral Small Molecule Inhibitor Of Anaplastic Lymphoma Kinase (ALK) And C-Met, In Children With Relapsed/Refractory Solid Tumors And Anaplastic Large Cell Lymphoma

For: Relapses/refractory solid tumors and anaplastic large cell lymphoma


A Single-Arm Multicenter Phase II Study preceded by Dose Evaluation to Investigate the Efficacy, Safety, and Tolerability of the BiTE® Antibody Blinatumomab (MT103) in Pediatric and Adolescent Patients with Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia

For: Relapsed or refractory B-precursor acute lymphoblastic leukemia


A phase II a, Single-Arm, Open-Label Study to Evaluate the Efficacy and Safety of the humanized anti-CD19 antibody MOR00208 in Adult Patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (B-ALL)

For: Relapsed or refractory B-cell acute lymphoblastic leukemia


Phase I/II a, 2-Part, Multi-Cancer, Single-Arm, Open-Label Study to determine the Safety, Tolerability and Pharmacokinetics of Oral Dabrafenib in Pediatric Subjects Aged 1 Month to < 18 Years with Advance BRAF V600-Mutation Positive Solid Tumors

For: Solid tumors, giomas, hystiosytosis


A Phase II Placebo-Controlled Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Tumor (SCUSF 0901; ACCL0922) Site CTEP ID= TN024

For: Previously treated brain tumors


A Phase II Randomized Trial of Lenalidomide (NSC#703813, IND#70116) in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytoma and Optic Pathway Gliomas

For: Recurrent, refractory or progressive brain tumors


Phase II Trial of Response-Based Radiation Therapy for Patients With Localized Central Nervous System Germ Cell Tumors

For: Newly diagnosed localize primary NGGCT or newly diagnosed localized germinoma


A Phase II, Randomized, Double-Blind, Placebo-Controlled Study to Examine the Effects of DAS181 in Immunocompromied Subjects with Lower Respiratory Tract ParaInfluenza (PIV) Infection on Supplemental Oxygen

For: Parainfluenza virus in immunocompromised patients


A Pilot Study of Decitabine and Vorinostat with Chemotherapy for Relapsed ALL (TACL protocol T2009-003)

For: Relapsed ALL


A Phase 2/3 multicenter, Open-Label, Multicohort, Two-Part Study Evaluating the Pharmacokinetics (PK), Safety, and Antiviral Activity of Elvitegravir (EVG) Administered with a Background Regimen (BR) Containing a Ritonavir-Boosted Protease Inhibitor (PI/r) in HIV-1 Infected, Antiretroviral Treatment-Experienced Pediatric Subjects

For: HIV


A Phase 1/2 Trial of Brentuximab Vedotin (SGN35, IND#117117) in Combination with Gemcitabine for Pediatric and Young Adult Patients with Relapsed or Refractory Hodgkin Lymphoma (AHOD1221)

For: Relapsed or Refractory Hodgkin Lymphoma


A Phase I, Open-Label, Dose Escalation Study of LDK378 in Pediatric Patients with Malignancies That Have a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)

For: Refractory or progressive malignancies


A Phase 2 Study of Ipilimumab in Children and adolescents (12 to < 18 years) with Previously Treated or Untreated, Unresectable Stage III or Stage IV Malignant Melanoma

For: Previously treated or untreated, unresectable Stage III or Stage IV malignant melanoma


An Open-label, Multicenter, Single-arm, Phase I Dose-escalation with Efficacy Tail Extension study of R05185426 in Pediatric Patients with Surgically Incurable and Unresectable Stage IIIC or Stage IV Melanoma Harboring BRAFV600

For: melanoma


A Phase I Trial of NECTAR (Nelarabine, Etoposide and Cyclophosphamide) in T-ALL Relapse: A Joint Study of TACL and POETIC

For: Leukemia


Phase I/II, Multi-Center, Open-Label Pharmacokinetic, Safety, Tolerability and Antiviral Activity of GSK1349572, a Novel Integrase Inhibitor, in Combination Regimens in HIV-1 Infected Infants, Children and Adolescents

For: Human Immunodeficiency Virus


A Phase I Dose Finding Study Of Panobinostat In Children With Refractory Hematologic Malignancies

For: Relapsed acute lymphoblastic leukemia [ALL], acute myelogenous leukemia [AML], Hodgkin disease [HD] and non-Hodgkin’s lymphomas [NHL]


Phase 1 and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma/ Brain Tumor

For: Gliomas


A Phase I /II Study of ABT-888, an Oral Poly (ADP-Ribose) Polymerase Inhibitor, and Concurrent Radiation Therapy, followed by ABT-888 and Temozolomide (TMZ), in Children with Newly Diagnosed Diffuse Intrinsic Pontine Gliomas (DIPG)

For: newly diagnosed diffuse intrinsic pontine gliomas (DIPG)


Phase II Study of Peginterferon Alfa-2B (PEGIntron) for Pediatric Patients with Unresectable or Recurrent Craniopharyngioma

For: Unresectable or recurrent craniopharyngioma


A Phase II Study of Everolimus for Recurrent or Progressive Low Grade Gliomas in Children

For: Recurrent or progressive low grade gliomas (brain tumor)


A Randomized, Double-Blinded, Placebo-Controlled, Multi-Institutional Phase II.5 Study of Saracatinib (AZD0530), a Selective Src Kinase Inhibitor, In Patients with Recurrent Osteosarcoma Localized to the Lung

For: recurrent osteosarcoma


A Phase II Study of Hypofractionated Stereotactic Radiotherapy in the Treatment of Metastatic Pediatric Sarcomas of Bony Sites

For: Metastatic sarcomas