A long-term follow-up study of subjects with β-thalassemia or sickle cell disease treated with study product (CTX001)
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).
About the study
Principal investigator
Dr. Hayley Merkeley
MD, FRCPC, Co-Director, Residency Training Program, Hematology, UBC, Clinical Assistant Professor, Department of Medicine, UBC, Hematologist, St Paul’s Hospital, and Vancouver General Hospital
Area(s) of medical focus
Blood & Blood Disorders
Condition(s)
Bleeding disorders
Period of enrollment
2022-08-02 - 2029-12-31
Participating sites
St. Paul's Hospital
Purpose of trial
The purpose of the study is to evaluate the long-term safety and efficacy of CTX001 in patients who underwent stem cell transplant with CTX001 for the management of transfusion-dependent Beta-Thalassemia (TDT).
CTX001 is an investigational drug; “investigational” means the drug is not yet approved by Health Canada, the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA) in the European Union or health authorities in other countries and is still being tested for safety and effectiveness.
Eligibility
You may be able to participate in this study at this institution if you:
- Received an infusion of CTX001
- Are able to understand and sign this informed consent form
There are no exclusion criteria for this study.
Contact information
Kelsi Sorensen, Main Study Coordinator
Tathiana Ruiz, Backup Study Coordinator
Rachel Despotovic, Research Manager
Researcher
Co-Investigators
Dr. Shannon Jackson, Dr. Heather Leitch
Additional Information
Study Type
Observational