A Long-term Follow-up Study in Patients who Received CTX001
Research type
Research Study
Full title
A Long-term Follow-up Study of Subjects With β thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
IRAS ID
255036
Contact name
Clinical Trials and Medical Information
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-002935-88
Clinicaltrials.gov Identifier
Duration of Study in the UK
17 years, 9 months, 28 days
Research summary
This study is designed to evaluate long term safety of a new investigational therapy named CTX001, in patients who received CTX001 for the treatment of Transfusion-dependent Beta-Thalassemia (TDT) or severe Sickle Cell Disease (SCD) during a previous study. During this study the participants will not receive CTX001 or other investigational product.
Participants will need to visit the study site every 3 months for the first 3 years, twice per year for the years 4 and 5 and then once per year for a duration of 15 years after the CTX001 administration done in the previous study. If the participant is unable or unwilling to come in for a scheduled site visit, the visit will be completed by telemedicine, and will include a visit to the participant’s home from a home health nurse followed by a conversation between the participant and investigator (i.e., in person, phone, or video conference) within 1 week of the home visit. During the site visits the participants will have abbreviated physical examinations, blood tests will be done. Participants with TDT will have liver and cardiac magnetic resonance imaging (MRI) done during this study.
Up to 90 participants will participate in this study around the world.
Participants that completed the parent study, including the 2 years follow-up after the CTX001 infusion, will be in the study for about 13 years.
Participants will be followed for a total of 15 years after the CTX infusion.REC name
Scotland B REC
REC reference
20/SS/0072
Date of REC Opinion
9 Jul 2020
REC opinion
Favourable Opinion