CTX001-121_Phase 1/2 study_SCD_Pro V2.0
Research type
Research Study
Full title
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
IRAS ID
251122
Contact name
Jesus de la Fuente Pereda
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-001320-19
Clinicaltrials.gov Identifier
18143, IND Number
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
This open-label study to evaluate the safety and efficacy of a single dose of an investigational gene-editing therapy (CTX001) in treating severe Sickle Cell Disease (SCD), an inherited blood disease that affects multiple organs with acute and chronic complications. CTX001 is a cellular product made using the participants’ own blood-forming stem cells edited through CRISPR-Cas9 technology. CTX001 aims to increase fetal Haemoglobin production with the goal of alleviating the effects of SCD. The initial study will enroll 12 adults (18-35 years), with the potential to expand to 45 total. The study involves 4 stages:
Stage 1 (~2-4months): Participants will sign an informed consent followed by screening procedures. Once eligibility is confirmed, participants may require several weeks of red blood cell transfusions in preparation for Stage 2.
Stage 2 (~2-4months): Participants will receive plerixafor to release the blood stem cells from the bone marrow into the blood, which are collected and sent to a manufacturing facility to create CTX001 (~8 wks). Additional unedited cells will be collected and stored at the study site to be used as for safety purposes. Participants will again receive transfusions to prepare for stem cell transplant procedures.
Stage 3 (~1 month): CTX001 must meet minimum target dose and editing requirements to be approved for release to the site. Once CTX001 is received by the site, the participant will receive chemotherapy, followed by CTX001 infusion through a central venous catheter by qualified site personnel. Infusion procedures are outlined in the study protocol and will be detailed in a separate manual supplied to all study sites.
Stage 4 (~ 2yrs follow-up): After CTX001 infusion, participants will be closely monitored in-patient until stable for discharge. Follow-up will continue for a total of 2 years post-CTX001, after which participants will be asked to continue in a separate follow-up study.
REC name
Scotland B REC
REC reference
18/SS/0106
Date of REC Opinion
28 Sep 2018
REC opinion
Favourable Opinion