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

Vertex Pharmaceuticals

Status and phase: Invitation-only; Phase 3

Conditions: Thalassemia; Beta-Thalassemia; Hemoglobinopathies; Genetic Diseases, Inborn; Sickle Cell Anemia; Sickle Cell Disease; Hematologic Diseases

Treatments: Biological: CTX001

Study type: Interventional

Funder types: Industry

Identifiers

NCT04208529

VX18-CTX001-131

2024-512654-19-00 (Other Identifier)

Details and patient eligibility

About

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), 161(NCT05477563),171 (NCT05951205) (severe sickle cell disease [SCD] studies).

Enrollment: 160 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
• Participants must have received CTX001 infusion in a parent study

Exclusion criteria

• There are no exclusion criteria

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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160 participants in 1 patient group

CTX001

Experimental group

Description:

All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151, CTX001-161 and CTX001-171) after CTX001 infusion will be asked to participate in this long-term follow-up study.

Treatment:

Biological: CTX001