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NCT05329649 · ClinicalTrials.gov registry record · Phase 3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
A Phase 3 study of Sickle Cell Disease and Hemoglobinopathies, sponsored by Vertex Pharmaceuticals Incorporated.
- Active
- Registry status
- Phase 3
- Development phase
- 13
- Enrollment target
- 7
- Study locations
NCT05329649 is a Phase 3 study of Sickle Cell Disease and Hemoglobinopathies that is active but no longer recruiting, run by Vertex Pharmaceuticals Incorporated. The registered enrollment target is 13 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (96% lower). The trial reports 7 study locations across 4 states.
The verdict
NCT05329649, a Phase 3 study of Sickle Cell Disease and Hemoglobinopathies, is active but no longer recruiting, sponsored by Vertex Pharmaceuticals Incorporated.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 13 participants
- Enrollment target
- 7
- Study locations
Study Summary
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Conditions Studied
Interventions
- BIOLOGICAL CTX001
Study Locations (7)
Other
- University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology - Düsseldorf
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Rome
- St.Mary's Hospital - Haematology Dept - London
Tennessee
- St. Jude Children's Research Hospital - Memphis
- TriStar Medical Group Children's Specialists - Pediatric Oncology - Nashville
North Carolina
- Levine Children's Hospital - Hematology - Charlotte
Pennsylvania
- The Children's Hospital of Philadelphia - Hematology - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2022-05-02 |
| Est. Completion | 2026-05-31 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05329649
The ClinicalTrials.gov registry entry for NCT05329649 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 13 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (96% lower). The listed sponsor is Vertex Pharmaceuticals Incorporated, which has 191 total studies on file at ClinicalTrials.gov.
The record links to 5 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which CTX001 is the first listed.
NCT05329649 reports 7 study locations spanning 4 distinct geographic areas - top geographies include Other, Tennessee, North Carolina.
Frequently Asked Questions
What is clinical trial NCT05329649 about?
NCT05329649 is a clinical study titled "Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)". This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
What is the current status of trial NCT05329649?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 13 participants. The study started on 2022-05-02. Estimated completion is 2026-05-31.
What conditions does trial NCT05329649 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Hemoglobinopathies, Hematological Diseases, Hydroxyurea Failure, Hydroxyurea Intolerance.
What interventions are being tested in trial NCT05329649?
The interventions under investigation include: CTX001 (BIOLOGICAL).
Who is sponsoring clinical trial NCT05329649?
This trial is sponsored by Vertex Pharmaceuticals Incorporated, which has 191 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05329649 being conducted?
This trial has 7 study locations across North Carolina, Pennsylvania, Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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