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NCT05477563 · ClinicalTrials.gov registry record · Phase 3
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
A Phase 3 study of Sickle Cell Disease and Sickle Cell Anemia, sponsored by Vertex Pharmaceuticals Incorporated.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 26
- Enrollment target
- 6
- Study locations
NCT05477563 is a Phase 3 study of Sickle Cell Disease and Sickle Cell Anemia that is actively recruiting participants, run by Vertex Pharmaceuticals Incorporated. The registered enrollment target is 26 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (91% lower). The trial reports 6 study locations across 4 states.
The verdict
NCT05477563, a Phase 3 study of Sickle Cell Disease and Sickle Cell Anemia, is actively recruiting participants, sponsored by Vertex Pharmaceuticals Incorporated.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 26 participants
- Enrollment target
- 6
- Study locations
Study Summary
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or 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.
Conditions Studied
Interventions
- BIOLOGICAL CTX001
Study Locations (6)
Other
- University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology - Düsseldorf
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Rome
- King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology - Al Mathar Ash Shamali
New York
- New York Presbyterian Hospital - Morgan Stanley Children's Hospital - New York
North Carolina
- Levine Children's Hospital - Hematology - Charlotte
Tennessee
- TriStar Medical Group Children's Specialists - Pediatric Oncology - Nashville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2022-08-02 |
| Est. Completion | 2027-06-09 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05477563
The ClinicalTrials.gov registry entry for NCT05477563 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 26 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 (91% lower). The listed sponsor is Vertex Pharmaceuticals Incorporated, which has 191 total studies on file at ClinicalTrials.gov.
The record links to 7 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which CTX001 is the first listed.
NCT05477563 reports 6 study locations spanning 4 distinct geographic areas - top geographies include Other, New York, North Carolina.
Frequently Asked Questions
What is clinical trial NCT05477563 about?
NCT05477563 is a clinical study titled "Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease". This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or 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 NCT05477563?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 26 participants. The study started on 2022-08-02. Estimated completion is 2027-06-09.
What conditions does trial NCT05477563 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Sickle Cell Anemia, Thalassemia, Hematologic Diseases, Hemoglobinopathies.
What interventions are being tested in trial NCT05477563?
The interventions under investigation include: CTX001 (BIOLOGICAL).
Who is sponsoring clinical trial NCT05477563?
This trial is sponsored by Vertex Pharmaceuticals Incorporated, which has 191 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05477563 being conducted?
This trial has 6 study locations across New York, North Carolina, Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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