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NCT05356195 · ClinicalTrials.gov registry record · Phase 3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
A Phase 3 study of Thalassemia and Hematologic Diseases, sponsored by Vertex Pharmaceuticals Incorporated.
- Active
- Registry status
- Phase 3
- Development phase
- 16
- Enrollment target
- 6
- Study locations
NCT05356195 is a Phase 3 study of Thalassemia and Hematologic Diseases that is active but no longer recruiting, run by Vertex Pharmaceuticals Incorporated. The registered enrollment target is 16 participants, below the 89-participant average among 19 other Thalassemia trials with a reported enrollment target (82% lower). The trial reports 6 study locations across 2 states.
The verdict
NCT05356195, a Phase 3 study of Thalassemia and Hematologic Diseases, is active but no longer recruiting, sponsored by Vertex Pharmaceuticals Incorporated.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 16 participants
- Enrollment target
- 6
- Study locations
Study Summary
This is a single-dose, open-label study in pediatric participants with TDT. 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 (6)
Other
- Hospital for Sick Children - Hematology - Toronto
- 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
- Great Ormond Street Hospital for Children - London
- St.Mary's Hospital - Children's Clinical Research Facility - London
Tennessee
- TriStar Medical Group Children's Specialists - Pediatric Oncology - Nashville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2022-05-03 |
| Est. Completion | 2026-05-31 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05356195
The ClinicalTrials.gov registry entry for NCT05356195 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 16 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 89-participant average among 19 other Thalassemia trials with a reported enrollment target (82% 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 Thalassemia appearing as the primary indexed condition, and to 1 intervention - of which CTX001 is the first listed.
NCT05356195 reports 6 study locations spanning 2 distinct geographic areas - top geographies include Other, Tennessee.
Frequently Asked Questions
What is clinical trial NCT05356195 about?
NCT05356195 is a clinical study titled "Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)". This is a single-dose, open-label study in pediatric participants with TDT. 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 NCT05356195?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 16 participants. The study started on 2022-05-03. Estimated completion is 2026-05-31.
What conditions does trial NCT05356195 study?
This clinical trial studies the following conditions: Thalassemia, Hematologic Diseases, Hemoglobinopathies, Beta-Thalassemia, Genetic Diseases, Inborn.
What interventions are being tested in trial NCT05356195?
The interventions under investigation include: CTX001 (BIOLOGICAL).
Who is sponsoring clinical trial NCT05356195?
This trial is sponsored by Vertex Pharmaceuticals Incorporated, which has 191 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05356195 being conducted?
This trial has 6 study locations across Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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