Home›Trial›NCT04208529
NCT04208529Phase 3ENROLLING BY INVITATION

A Long-term Follow-up Study in Participants Who Received CTX001

Study Summary

This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).

Conditions
Beta-ThalassemiaThalassemiaSickle Cell DiseaseHematologic DiseasesHemoglobinopathiesGenetic Diseases, InbornSickle Cell Anemia
Interventions / Drugs
CTX001
View on ClinicalTrials.gov ↗
Sponsor
VRTX
Vertex Pharmaceuticals
Gene Therapy
F22/100
Study Details
Enrollment160 participants
Primary Completion—
Lead SponsorVertex Pharmaceuticals Incorporated
Data Retrieved2026-04-11 02:55:58.035356+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

Current status: ENROLLING BY INVITATION.

Enrolled: 160 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.

VRTX Full Pipeline →All Companies →
Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.