Phase 2 N=15 Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Juvenile Myelomonocytic Leukemia
Primary: Determine Probability of Long-term Disease Free Survival in JMML — 67 Percentage of participants
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Phase 2 N=30 Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia
Primary: Percent Probability of Event-free Survival (EFS) — 83; 22 percent probability
Phase 2 N=19 Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Juvenile Neuronal Ceroid Lipofuscinosis
Primary: Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose — 17; 19 Participants — p=0.21
Phase 2 N=46 Prospective Study of Mylotarg and G-CSF in Acute Myeloid Leukemia Treatment
Novo Acute Myeloid Leukemia
Primary: Complete Remission of the Disease — NA; 18; 16; NA Participants
Phase 2 N=10 Sunitinib in Treating Patients With Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
Chronic Myelomonocytic Leukemia · de Novo Myelodysplastic Syndromes · Myelodysplastic Syndromes
Primary: Overall Response Rate (Complete Response, Partial Response, or Hematologic Improvement) Defined by the International Working Group Criteria — 10 percentage of participants
Phase 4 N=10 Abatacept in Juvenile Dermatomyositis
Dermatomyositis
Primary: Number of Patients Meeting the Definition of Improvement (DOI) at Week 24: at Least 3 of 6 Core Set Measures (CSM) Improved by ≥ 20% With no More Than 2 CSM Worsening by…
Phase 3 N=139 Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis
Juvenile Dermatomyositis
Primary: Responder Status Defined as 20% Improvement in at Least 3 Core Set Variables With no More Than 1 of the Remaining Variables, (Muscle Strength Excluded), Worsened by >…
Phase 3 N=205 Combination Chemotherapy in Treating Young Patients With Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndromes
Childhood Acute Basophilic Leukemia · Childhood Acute Eosinophilic Leukemia · Childhood Acute Erythroleukemia (M6)
Primary: Event-free Survival (EFS) at 3 Years — 90.1 percentage
Phase 2 N=9 Tagraxofusp (SL-401) in Participants With Chronic Myelomonocytic Leukemia (CMML) and Myelofibrosis (MF)
Myelofibrosis · Chronic Myelomonocytic Leukemia
Primary: Number of Participants With Dose-limiting Toxicities (DLTs) — 0; 0; 0; 0 participants
Phase 2 N=57 A Phase 2 Trial of MLN8237 in Adult Participants With Acute Myelogenous Leukemia and High-Grade Myelodysplastic Syndrome
Acute Myelogenous Leukemia · High-Grade Myelodysplastic Syndrome
Primary: Best Overall Response Rate (ORR) Based on Investigator's Assessment — 6; 0; 1; 0 participants
Phase 2 N=34 Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia
Leukemia · Myelodysplastic Syndromes · Childhood Acute Myeloid Leukemia in Remission
Primary: Incidence of Engraftment — 100; 93; 100 Percentage of participants
Phase 2 N=4 Wilm's Tumor 1 (WT1) Peptide Vaccine for High Risk Hematologic Malignancy
Myelodysplastic Syndrome · Acute Myeloid Leukemia (AML) · Chronic Myeloid Leukemia (CML)
Primary: Cellular Immune Response — 4 participants
Phase 3 N=1,070 Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia
Leukemia
Primary: Event-free Survival at 3 Years — 46.9; 53.1; 50.0 percentage of participants
Phase 3 N=108 Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia
Childhood Acute Promyelocytic Leukemia With PML-RARA · Myeloid Neoplasm
Primary: Event-free Survival (EFS) — 95.4; 82.9 Percentage of participants
Phase 2 N=158 Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia
Leukemia
Primary: Overall Survival (OS) — 45.9 Percentage of participants
Phase 2 N=27 Revlimid in Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome
Acute Myelogenous Leukemia · Myelodysplastic Syndrome
Primary: Number of Participants With a Response (Complete Remissions (CR), Complete Remissions With Incomplete Platelet Recovery [CRp] and Partial Responses) — 1; 1; 0 participants
Phase 2 N=151 Fludarabine and Cytarabine in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS)
Leukemia · AML · MDS
Primary: Participants With a Complete Response — 34 Participants
Phase 2 N=31 Ruxolitinib and Lenalidomide for Patients With Myelofibrosis
Myeloproliferative Diseases
Primary: Participants With Objective Response — 7 Participants
Phase 1 N=5 Volasertib in Combination With Azacitidine in Japanese Patients With Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
Myelodysplastic Syndromes · Leukemia, Myelomonocytic, Chronic
Primary: Number of Participants With Dose Limiting Toxicities (DLTs) in the First Cycle for the Determination of the Maximum Tolerated Dose (MTD) — 2 Participants
Phase 2 N=50 A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib
Myelomonocytic Leukemia
Primary: The Maximum Tolerated Dose (MTD) of Ruxolitinib for the Treatment of Myelomonocytic Leukemia (CMML) — 40 mg
Phase 2 N=10 PD-1 Inhibition in Advanced Myeloproliferative Neoplasms
Chronic Phase Myelofibrosis · Primary Myelofibrosis · Post-essential Thrombocythemia Myelofibrosis
Primary: European Leukemia Net -International Working Group (ELN-IWG) Criteria — 0 Participants
Phase 2 N=6 Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation
Accelerated/Blast-phase Myeloproliferative Neoplasm · Chronic-phase Myelofibrosis · IDH2 Mutation
Primary: Number of MPN Participants With Response — 2 Participants
Phase 2 N=31 Azacitidine and Lenalidomide for Acute Myeloid Leukemia
Leukemia, Myeloid, Acute
Primary: Phase I Only - Maximum Tolerated Dose (MTD) as Measured by Dose-limiting Toxicities (DLTs) — 1; 0; 0 dose-limiting toxicities
Phase 2 N=35 3-AP and Fludarabine in Treating Patients With Myeloproliferative Disorders, Chronic Myelomonocytic Leukemia, or Accelerated Phase or Blastic Phase Chronic Myelogenous Leukemia
Accelerated Phase Chronic Myelogenous Leukemia · Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative · Blastic Phase Chronic Myelogenous Leukemia
Primary: Response Rate Including Complete Response, Partial Response, and Hematological Improvement Assessed by Blood Cell Counts, Number of Blasts in Bone Marrow, and Clinical…
Phase 1 N=24 AC220 for Children With Relapsed/Refractory ALL or AML
Lymphoblastic Leukemia, Acute, Childhood · Myelogenous Leukemia, Acute, Childhood
Primary: The Dose of AC220 That is Safe and Biologically Active When Given in Sequential Combination With Ara-C/Etoposide — 0; 3; 1; 4 Participants
Phase 2 N=78 Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement
Acute Leukemia of Ambiguous Lineage · B Acute Lymphoblastic Leukemia · Mixed Phenotype Acute Leukemia
Primary: Tolerability of Azacitidine in Combination With Interfant-06 Standard Chemotherapy in Evaluable Infant Patients With Newly Diagnosed ALL With KMT2A Gene Rearrangement…
N/A N=16 Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies
Myeloid Hematological Malignancies
Primary: Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients. — 0.923; 0.923; 0.923 probability of overall survival
Phase 2 N=40 Decitabine and Gemtuzumab Ozogamicin in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (H-R MDS)
Acute Myelogenous Leukemia · Myelodysplastic Syndrome
Primary: Number of Participants With a Complete Response — 10 Participants
Phase 1 N=15 A Phase I Study of 5-Azacytidine in Combination With Chemotherapy for Children With Relapsed or Refractory ALL or AML
Lymphoblastic Leukemia, Acute, Childhood · Myelogenous Leukemia, Acute, Childhood
Primary: Number of Participants Who Experienced a Dose Limiting Toxicity (DLT) — 0; 0; 12; 2 Participants
Phase 2 N=19 Pharmacokinetics-based Mycophenolate Mofetil Dosing for GVHD Prevention
Allogeneic Blood and Marrow Transplantation (BMT) · Graft Versus Host Disease
Primary: Number of Participants With Grade ≥3 Toxicities Scored According to the CTCAE Version 4.0. — 0 participants