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A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Recruiting
Trial ID: NCT03745287
Purpose
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with
severe sickle cell disease (SCD). 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 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
Stanford Investigator(s)
Eligibility
Key Inclusion Criteria:
- Diagnosis of severe sickle cell disease as defined by:
- Documented severe sickle cell disease genotype
- History of at least two severe vaso-occlusive crisis events per year for the previous
two years prior to enrollment
- Eligible for autologous stem cell transplant as per investigators judgment
Key Exclusion Criteria:
- An available 10/10 human leukocyte antigen (HLA)-matched related donor
- Prior hematopoietic stem cell transplant (HSCT)
- Clinically significant and active bacterial, viral, fungal, or parasitic infection
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