Phase 2 N=15 Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Anemia, Diamond-Blackfan
Primary: Participants That Responded to Eltrombopag — 1; 14 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=11 Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 participants
Phase 2 N=55 Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
Diamond Blackfan Anemia · Blackfan Diamond Syndrome · DBA
Primary: Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia — 2; 5; 36; 0 Participants
Phase 2 N=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=14 Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis
Anemia · Primary Myelofibrosis · Secondary Myelofibrosis
Primary: Best Overall Response Rate as Determined by International Working Group Criteria — 28.6 percentage of patients with CR, PR or CI
Phase 2 N=27 Danazol for Genetic Bone Marrow and Lung Disorders
Aplastic Anemia
Primary: Number of Patients Having Attenuation of Accelerated Telomere Attrition — 12 Participants
Phase 2 N=102 Darbepoetin Administration to Preterm Infants
Infant, Newborn
Primary: Number of Transfusions During Hospitalization — 1.2; 1.2; 2.4 number of transfusions
Phase 3 N=147 Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
MDS
Primary: Percentage of Participants With at Least One Red Blood Cell (RBC) Transfusion During the Double-blind Treatment Period — 59.2; 36.1 percentage of participants — p=0.008
Phase 2 N=34 Eltrombopag for Moderate Aplastic Anemia
Moderate Aplastic Anemia · Unilineage Bone Marrow Failure Disorders
Primary: Proportion of Drug Responders — 17 Participants
Phase 2 N=45 Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Aplastic Anemia · Leukemia · Myelodysplastic Syndrome
Primary: Successful Neutrophil Engraftment — 44; 1 Participants
Phase 2 N=6 Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia
Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
Phase 2 N=43 A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Anemia, Aplastic · Anemia, Hypoplastic · Thrombocytopenia
Primary: The Portion of Drug Responders as Defined by Hematologic Improvements — 17 Participants
Phase 2 N=31 Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 0; 0; 0 Participants
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
Phase 2 N=30 Sirolimus for Autoimmune Disease of Blood Cells
Autoimmune Pancytopenia · Autoimmune Lymphoproliferative Syndrome (ALPS) · Evans Syndrome
Primary: Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus — 1 participants
Phase 2 N=1 Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
Fanconi Anemia
Primary: Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF — 1 participants
Phase 2 N=12 Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Early Phase 1 N=5 RHD Genotype Matched Red Cells for Anti-D
Sickle Cell Disease · Anti-D Antibodies
Primary: Anti-D Recurrence — 0; 5 Participants
Phase 2 N=25 Defibrotide TMA Prophylaxis Pilot Trial
Thrombotic Microangiopathies
Primary: Percent of Total Doses of Defibrotide That Were Missed [Feasibility] — 0.7 percentage of missed doses
Phase 2 N=14 Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 1; 0; 0; 0 Participants
Phase 2 N=30 A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)
Myelodysplastic Syndromes · Thrombocytopenia
Primary: Number of Participants With Response Between Weeks 16 and 20 — 11 Participants
Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
N/A N=29 Calcium Absorption in Patients With Rothmund-Thomson Syndrome
Rothmund-Thomson Syndrome
Primary: Bone Density (Low Areal Bone Mineral Density (aBMD)) — -1.3; -0.9; -2.4; -1.2 z-score
Phase 2 N=12 Use of ACTIMMUNE in Patients With ADO2
Autosomal Dominant Osteopetrosis Type 2
Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Phase 2 N=14 Pilot Study of Metformin for Patients With Fanconi Anemia
Fanconi Anemia
Primary: Hematologic Response — 4 Participants
Phase 2 N=48 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Myelodysplastic Syndrome · Aplastic Anemia
Primary: Overall Response — 8; 8; 4 participants
Phase 2 N=47 Alemtuzumab to Treat Severe Aplastic Anemia
Severe Aplastic Anemia, Refractory · Severe Aplastic Anemia, Relapse
Primary: Number of Participants With Hematological Response at 6 Months — 0; 18 Participants
Phase 3 N=1,683 Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Thalassemia · Sickle Cell Disease · Diamond Blackfan Anemia
Primary: Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events — 0; 0; 1; 4 Participants
Phase 3 N=4 Anemia Studies in CKD: Erythropoiesis Via a Novel PHI Daprodustat - Pediatric (ASCEND-P)
Anaemia
Primary: Number of Participants With Any Adverse Events (AEs) and Serious Adverse Events (SAEs) — 2; 2; 1; 0 Participants