Bone Marrow Failure Syndromes
16
3
4
7
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.3%
1 terminated out of 16 trials
87.5%
+0.9% vs benchmark
13%
2 trials in Phase 3/4
43%
3 of 7 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 7 completed trials
Clinical Trials (16)
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Investigation of the Genetics of Hematologic Diseases
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Fludarabine Based RIC for Bone Marrow Failure Syndromes
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
Methadone as an Alternative Treatment for Children Underdoing HSCT
UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
Nandrolone Decanoate in the Treatment of Telomeropathies
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Memory T-cell Infusion to Improve Immunity After TCR-alpha/Beta Depleted Hematopoietic Stem Cell Transplantation
Unrelated Donor Transplant for Malignant and Non-Malignant Disorders
Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCT
Identifying Characteristics of Bone Marrow Failure Syndromes