Safety and Efficacy of CTX001 in Pediatric Subjects With TDT
Research type
Research Study
Full title
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
IRAS ID
307875
Contact name
Josu de la Fuente Pereda
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2021-002172-39
Duration of Study in the UK
3 years, 9 months, 31 days
Research summary
This Phase 3, single-dose, open-label study in paediatric subjects with TDT. This study is being done to learn more about the safety and efficacy of CTX001 in participants with transfusion dependent β-Thalassemia (TDT)and will include a minimum of 8 participants, 2 through 11 years of age, inclusive.Enrolment will be staggered based on Data Monitoring Committee (DMC) review of available engraftment, safety, and efficacy data.The study will be conducted in 4 stages:
Stage 1: Screening period (1 to 3 months) Participants will sign an informed consent followed by screening procedures. After signing the informed consent form, screening tests are performed to see if participant can be in the study ("eligible")
Stage 2: Blood stem cell collection and study drug manufacture (approximately 2-3 months) Participants will receive a combination of medications called granuloctye-colony stimulating factor (G-CSF) and plerixafor in order to release the blood stem cells from participants bone marrow into bloodstream (a process called mobilization) so that they can be collected through a process called apheresis.The cells collected from participant's bloodstream will be used to create the Study Product.
Stage 3: Chemotherapy conditioning and Study Product infusion (1 month)
Stage 4: Follow-up Through Engraftment and Up To 24 Months After CTX001 infusionREC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0883
Date of REC Opinion
11 May 2022
REC opinion
Further Information Favourable Opinion