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NCT01896102 · ClinicalTrials.gov registry record · Phase 2
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
A Phase 2 study of Cerebral Adrenoleukodystrophy (CALD), sponsored by Genetix Biotherapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 32
- Enrollment target
- 8
- Study locations
NCT01896102: Completed Phase 2 study of Cerebral Adrenoleukodystrophy (CALD), sponsored by Genetix Biotherapeutics.
NCT01896102 is a Phase 2 study of Cerebral Adrenoleukodystrophy (CALD) that has completed, run by Genetix Biotherapeutics. The registered enrollment target is 32 participants, below the 75-participant average among 3 other Cerebral Adrenoleukodystrophy (CALD) trials with a reported enrollment target (57% lower). The trial reports 8 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01896102, a Phase 2 study of Cerebral Adrenoleukodystrophy (CALD), has completed, sponsored by Genetix Biotherapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 32 participants
- Enrollment target
- 8
- Study locations
Study Summary
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were 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 were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.
Primary Outcome
The 6 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 was defined as: alive at 24 months post-infusion; had not developed any of the MFDs by 24 months post-infusion; had not received rescue cell administration or allo-HSCT by 24 months post-infusion; and had not withdrawn from the study or had not been lost to follow-up by 24 months post-infusion. Percent
Conditions Studied
Interventions
- GENETIC Lenti-D Drug Product (eli-cel)
Study Locations (8)
Other
- Medeos SRL - Buenos Aires
- Hôpital Bicêtre - Le Kremlin-Bicêtre
- University of Leipzig - Leipzig
- Great Ormond Street Hospital for Children NHS Foundation Trust - London
California
- Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center - Los Angeles
Massachusetts
- Boston Children's Hospital/Massachusetts General Hospital - Boston
Minnesota
- University of Minnesota - Minneapolis
South Australia
- Women and Children's Hospital - North Adelaide
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2013-08-21 |
| Est. Completion | 2021-03-26 |
| Phase | Phase 2 |
What the finished NCT01896102 record still lists
NCT01896102 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 75-participant average among 3 other Cerebral Adrenoleukodystrophy (CALD) trials with a reported enrollment target (57% lower).
The record links to 1 condition, with Cerebral Adrenoleukodystrophy (CALD) appearing as the primary indexed condition, and to 1 intervention - of which Lenti-D Drug Product (eli-cel) is the first listed.
NCT01896102 lists 8 locations in 5 states (Other, California, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT01896102 about?
NCT01896102 is a clinical study titled "A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)". This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blo...
What is the current status of trial NCT01896102?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 32 participants. The study started on 2013-08-21. Estimated completion is 2021-03-26.
What conditions does trial NCT01896102 study?
This clinical trial studies the following conditions: Cerebral Adrenoleukodystrophy (CALD).
What interventions are being tested in trial NCT01896102?
The interventions under investigation include: Lenti-D Drug Product (eli-cel) (GENETIC).
Who is sponsoring clinical trial NCT01896102?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01896102 being conducted?
This trial has 8 study locations across California, Massachusetts, Minnesota, South Australia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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