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NCT03837483 · ClinicalTrials.gov registry record · Phase 3
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
A Phase 3 study of Wiskott-Aldrich Syndrome, sponsored by Fondazione Telethon.
- Active
- Registry status
- Phase 3
- Development phase
- 10
- Enrollment target
- 2
- Study locations
NCT03837483: Active Phase 3 study of Wiskott-Aldrich Syndrome, sponsored by Fondazione Telethon.
NCT03837483 is a Phase 3 study of Wiskott-Aldrich Syndrome that is active but no longer recruiting, run by Fondazione Telethon. The registered enrollment target is 10 participants, below the 108-participant average among 4 other Wiskott-Aldrich Syndrome trials with a reported enrollment target (91% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03837483, a Phase 3 study of Wiskott-Aldrich Syndrome, is active but no longer recruiting, sponsored by Fondazione Telethon.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 10 participants
- Enrollment target
- 2
- Study locations
Study Summary
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.
Conditions Studied
Interventions
- GENETIC OTL-103
Study Locations (2)
Georgia
- Children's Healthcare of Atlanta, Inc - Atlanta
Other
- Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) - Milan
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2019-01-21 |
| Est. Completion | 2027-09 |
| Phase | Phase 3 |
What the registry record for NCT03837483 still lists
NCT03837483 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 108-participant average among 4 other Wiskott-Aldrich Syndrome trials with a reported enrollment target (91% lower).
The record links to 1 condition, with Wiskott-Aldrich Syndrome appearing as the primary indexed condition, and to 1 intervention - of which OTL-103 is the first listed.
NCT03837483 reports a single indexed study location in Georgia, Other.
Frequently Asked Questions
What is clinical trial NCT03837483 about?
NCT03837483 is a clinical study titled "A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome". This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.
What is the current status of trial NCT03837483?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 10 participants. The study started on 2019-01-21. Estimated completion is 2027-09.
What conditions does trial NCT03837483 study?
This clinical trial studies the following conditions: Wiskott-Aldrich Syndrome.
What interventions are being tested in trial NCT03837483?
The interventions under investigation include: OTL-103 (GENETIC).
Who is sponsoring clinical trial NCT03837483?
This trial is sponsored by Fondazione Telethon, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03837483 being conducted?
This trial has 2 study locations across Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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