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NCT03852498 · ClinicalTrials.gov registry record · Phase 3
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
A Phase 3 study, sponsored by Genetix Biotherapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 35
- Enrollment target
NCT03852498: Completed Phase 3 study, sponsored by Genetix Biotherapeutics.
NCT03852498 is a Phase 3 study that has completed, run by Genetix Biotherapeutics. The registered enrollment target is 35 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03852498, a Phase 3 study, has completed, sponsored by Genetix Biotherapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 35 participants
- Enrollment target
Study Summary
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).
Primary Outcome
The MFDs consisted of loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement. Month-24 MFD-Free survival criteria defined as: alive at 24 months post infusion; have not developed any of the MFDs by 24 months post infusion; have not received rescue cell administration or allo-hematopoietic stem cell transplantation (HSCT) by 24 months post infusion; and have not withdrawn from the study or have not been lost to follo
Interventions
- GENETIC Lenti-D
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2019-01-24 |
| Est. Completion | 2023-07-24 |
| Phase | Phase 3 |
What the finished NCT03852498 record still lists
NCT03852498 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 1 intervention - of which Lenti-D is the first listed.
NCT03852498 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03852498 about?
NCT03852498 is a clinical study titled "A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)". The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells wil...
What is the current status of trial NCT03852498?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 35 participants. The study started on 2019-01-24. Estimated completion is 2023-07-24.
What interventions are being tested in trial NCT03852498?
The interventions under investigation include: Lenti-D (GENETIC).
Who is sponsoring clinical trial NCT03852498?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03852498's enrollment target sits among peer trials
35 1903rd of 2000 higher than 96 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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