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NCT05356195Phase 3ACTIVE NOT RECRUITING

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

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
Beta-ThalassemiaThalassemiaGenetic Diseases, InbornHematologic DiseasesHemoglobinopathies
Interventions / Drugs
CTX001
View on ClinicalTrials.gov ↗
Sponsor
VRTX
Vertex Pharmaceuticals
Gene Therapy
F22/100
Study Details
Enrollment16 participants
Primary Completion—
Lead SponsorVertex Pharmaceuticals Incorporated
Data Retrieved2026-04-11 02:55:58.035274+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 16 participants
Sponsor Catalyst Profile

Vertex Pharmaceuticals (VRTX) is the sponsoring company for this trial. BiotechSign currently grades this company F (22/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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