Osteopetrosis
16
1
2
7
Key Insights
Highlights
Success Rate
58% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
31.3%
5 terminated out of 16 trials
58.3%
-28.2% vs benchmark
6%
1 trials in Phase 3/4
71%
5 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 (16)
Longitudinal Study of Neurodegenerative Disorders
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
ACTIMMUNE in Intermediate Osteopetrosis
Outcomes of Patients With Osteopetrosis Weight-bearing Bone Fractures
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
T-cell Depleted Alternative Donor Transplantation
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
Reduced Intensity AlloTransplant For Osteopetrosis
Stem Cell Transplantation for Children Affected With Osteopetrosis
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
rhPTH Therapy for Low Turnover Bone Fragility
Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis