Bone Marrow Failure
18
5
7
3
Key Insights
Highlights
Success Rate
60% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 60/100
11.1%
2 terminated out of 18 trials
60.0%
-26.5% vs benchmark
0%
0 trials in Phase 3/4
100%
3 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (18)
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
European Rare Blood Disorders Platform (ENROL)
NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
T-cell Depleted Alternative Donor Transplantation
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
Identification of Autoantibodies and Autoantigens in the Cerebrospinal Fluid of Patients With Spinal Cord Trauma
CD34+ Stem Cell Infusion to Augment Graft Function
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)