Safety and Efficacy of CTX001 in Pediatric Subjects With SDC
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 Severe Sickle Cell Disease
IRAS ID
307706
Contact name
Josu de la Fuente Pereda
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2021-002173-26
Duration of Study in the UK
4 years, 5 months, 3 days
Research summary
This Phase 3, single-dose, open-label study in paediatric participants with severe
SCD and HU failure or intolerance. This study is being done to learn more about the safety and effectiveness of CTX001 in patients with severe SCD 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 (approximately 2-4 months) Participants will be asked to sign this informed consent form before any study tests or procedures are done. 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 Product manufacture (approximately 2-4 months) Participants will receive a medication called plerixafor in order to release the blood stem cells from participant's bone marrow into bloodstream (a process called mobilisation) 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 (approximately 1 month) Participant will be admitted to the hospital at the study site to prepare participant's body to receive the Study Product.
Stage 4: Follow-up (approximately 2 years after receiving Study Product).Data findings as of 01 June 2021 confirm 17 subjects with SCD and 27 subjects with TDT have received CTX001. A total of 44 subjects have received CTX001.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
22/LO/0392
Date of REC Opinion
5 Sep 2022
REC opinion
Further Information Favourable Opinion