Bone Marrow Failure Syndromes
15
4
4
7
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.7%
1 terminated out of 15 trials
87.5%
+1.0% 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: 86.5%
Based on 7 completed trials
Clinical Trials (15)
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
Investigation of the Genetics of Hematologic Diseases
Methadone as an Alternative Treatment for Children Underdoing HSCT
UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
Fludarabine Based RIC for Bone Marrow Failure Syndromes
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