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NCT03745287 · ClinicalTrials.gov registry record · Phase 2
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
A Phase 2 study, sponsored by Vertex Pharmaceuticals Incorporated.
- Completed
- Registry status
- Phase 2
- Development phase
- 63
- Enrollment target
NCT03745287 is a Phase 2 study that has completed, run by Vertex Pharmaceuticals Incorporated. The registered enrollment target is 63 participants.
The verdict
NCT03745287, a Phase 2 study, has completed, sponsored by Vertex Pharmaceuticals Incorporated.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 63 participants
- Enrollment target
Study Summary
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
Interventions
- BIOLOGICAL CTX001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 63 participants |
| Start Date | 2018-11-27 |
| Est. Completion | 2025-07-07 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03745287
The ClinicalTrials.gov registry entry for NCT03745287 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 63 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Vertex Pharmaceuticals Incorporated, which has 191 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which CTX001 is the first listed.
NCT03745287 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03745287 about?
NCT03745287 is a clinical study titled "A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease". This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
What is the current status of trial NCT03745287?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 63 participants. The study started on 2018-11-27. Estimated completion is 2025-07-07.
What interventions are being tested in trial NCT03745287?
The interventions under investigation include: CTX001 (BIOLOGICAL).
Who is sponsoring clinical trial NCT03745287?
This trial is sponsored by Vertex Pharmaceuticals Incorporated, which has 191 total clinical trials registered on ClinicalTrials.gov.
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