A Long-term Follow-up Study in Subjects Who Received CTX001
Sponsored by Vertex Pharmaceuticals Incorporated
About this trial
Last updated 2 years ago
Study ID
CTX001-131
Status
Enrolling by invitation
Type
Observational
Placebo
No
Accepting
2+ Years
All
Not accepting
Healthy Volunteers
Trial Timing
Started 4 years ago
What is this trial about?
This is a multi-site, observational study to evaluate the long-term safety and efficacy of
CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or VX21-CTX001-141
(transfusion-dependent β-thalassemia [TDT] studies) or Study CTX001-121 (NCT03745287) or
VX21-CTX001-151 (severe sickle cell disease [SCD] studies; NCT05329649).
What are the participation requirements?
Inclusion Criteria
- Subjects (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
- Subjects must have received CTX001 infusion in a parent study
Exclusion Criteria
- There are no exclusion criteria