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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 is a Phase 3 study that has completed, run by Genetix Biotherapeutics. The registered enrollment target is 35 participants.
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03852498
The ClinicalTrials.gov registry entry for NCT03852498 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genetix Biotherapeutics, which has 12 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Lenti-D is the first listed.
NCT03852498 reports 0 study locations.
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.
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