Cerebral Adrenoleukodystrophy (CALD)
6
1
3
2
Key Insights
Highlights
Success Rate
67% trial completion
Treatment Research
7 treatments under study, led by Lentid
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
16.7%
1 terminated out of 6 trials
66.7%
-19.9% vs benchmark
33%
2 trials in Phase 3/4
100%
2 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 2 completed trials
Clinical Trials (6)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Research Network
Common Treatments
- Lentid1
- Elivaldogene Autotemcel1
- Allohsct1
- Leriglitazone1
- No Intervention1
Active Sponsors
- Genetix Biotherapeutics Inc.5
- Center for International Blood and Marrow Transplant Research1
- Minoryx Therapeutics, S.L.1
Top Investigators
Hôpital Bicêtre
University of California, Los Angeles
bluebird bio, Inc.
Boston Children's Hospital
Massachusetts General Hospital
Related Conditions
- Adrenoleukodystrophy Ald2
- Xlinked Adrenoleukodystrophy Xald2