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NCT05477563Phase 3RECRUITING

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

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
Beta-ThalassemiaThalassemiaHematologic DiseasesGenetic Diseases, InbornHemoglobinopathiesSickle Cell DiseaseSickle Cell Anemia
Interventions / Drugs
CTX001
View on ClinicalTrials.gov ↗
Sponsor
VRTX
Vertex Pharmaceuticals
Gene Therapy
F22/100
Study Details
Enrollment26 participants
Primary Completion—
Lead SponsorVertex Pharmaceuticals Incorporated
Data Retrieved2026-04-11 02:55:58.035170+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

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 26 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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