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


An Open- Label, Dose Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescent Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 Mutations.

For: Refractory or refractory solid tumor


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 Study of Bortezomib and Vorinostat in Patients with Refractory or Relapsed MLL Rearranged Hematologic Malignancies

For: Relapsed or refractory Acute Lymphoblastic Leukemia (ALL), Biphenotypic/Mixed Lineage Leukemia (MLL), or Acute Myeloid Leukemia (AML)


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 or Progressive Central Nervous System Atypical Teratoid Rhabdoid Tumors (ATRT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnosed ATRT

For: Recurrent, 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 I, Open Label, Single-Dose, Non-Randomized Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban in Pediatric Patients

For: For patients at risk for blood clots


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


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