Shwachman-Diamond Syndrome
12
2
2
7
Key Insights
Highlights
Success Rate
70% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 74/100
25.0%
3 terminated out of 12 trials
70.0%
-16.5% vs benchmark
0%
0 trials in Phase 3/4
57%
4 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 (12)
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
Baby Detect : Genomic Newborn Screening
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
Diabetes/ Endocrine Surveillance in SDS
Shwachman Diamond Syndrome Registry and Study
Stem Cell Transplant for Hemoglobinopathy
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
Stem Cell Transplant for Bone Marrow Failure Syndromes
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment