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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: Active Phase 3 study of Thalassemia and Hematologic Diseases, sponsored by Vertex Pharmaceuticals Incorporated.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What the registry record for NCT05356195 still lists
NCT05356195 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 89-participant average among 19 other Thalassemia trials with a reported enrollment target (82% lower).
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 lists 6 locations in 2 states (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.
Learn More About Clinical Trials
Similar trials for Thalassemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05356195's enrollment target sits among peer trials
16 18th of 19 higher than 2 of 19 other Thalassemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Thalassemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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