Inherited Bone Marrow Failure Syndrome
5
4
4
0
Key Insights
Highlights
No notable highlights available yet
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 85/100
20.0%
1 terminated out of 5 trials
0.0%
-86.5% vs benchmark
0%
0 trials in Phase 3/4
0%
1 of 0 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Clinical Trials (5)
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases