Clinical trial
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the…
NCT03852498 · Genetix Biotherapeutics Inc., Industry · Completed
Key facts
| NCT number | NCT03852498 |
|---|---|
| Title | A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) |
| Official title | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) |
| Lead sponsor | Genetix Biotherapeutics Inc., Industry |
| Status | Completed |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Cerebral Adrenoleukodystrophy (CALD) |
| Intervention type | Genetic |
| Interventions | Lenti-D |
| Enrollment | 35 |
| Start date | Jan 24, 2019 |
| Primary completion | Jul 24, 2023 |
| Completion | Jul 24, 2023 |
| First posted | Feb 25, 2019 |
| Last update posted | May 24, 2024 |
| Countries | France, Germany, Italy, Netherlands, United Kingdom, United States |
At a glance
- 1–9 Study sites
- Yes Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: