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A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
Recruiting
Trial ID: NCT03655678
Purpose
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with
transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of
autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs)
using CTX001.
Official Title
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
Stanford Investigator(s)
Eligibility
Key Inclusion Criteria:
- Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by:
1. Documented homozygous β-thalassemia or compound heterozygous β-thalassemia
including β-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on
historical data, but a confirmation of the genotype using the study central
laboratory will be required before busulfan conditioning.
2. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions
in the prior 2 years before signing the consent or the last rescreening for
patients going through re-screening.
- Eligible for autologous stem cell transplant as per investigator's judgment.
Key Exclusion Criteria:
- A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is
available per investigator's judgement.
- Prior allo-HSCT.
- Subjects with associated α-thalassemia and >1 alpha deletion or alpha multiplications.
- Subjects with sickle cell beta thalassemia variant.
- Clinically significant and active bacterial, viral, fungal, or parasitic infection as
determined by the investigator.
- White blood cell (WBC) count <3 × 10^9/L or platelet count <50 × 10^9/L not related to
hypersplenism.
Other protocol defined Inclusion/Exclusion criteria may apply.
Intervention(s):
biological: CTX001
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305