Recruiting completed
Phase 2,
Phase 3
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
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.
ClinicalTrials.gov Identifier
Eligibility criteria
Sex
Age
12 Years - 35 Years
Phase
2, 3
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.
Trial details
Condition
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
Study duration
November 2018 - October 2024
Study type
Interventional
Intervention/Treatment
Biological : CTX001
Estimated enrollment
45
Interested in this trial?
About clinical trials
Volunteers who participate in clinical trials help make new medicines a reality. Clinical trials are carefully designed research studies in humans that evaluate the safety and efficacy of an investigational medicine.
If you’re interested in participating in a Vertex clinical trial, we encourage you to speak with your health care provider first about what’s involved and your eligibility.
Location
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Recruiting completed1Lucille Packard Children's Hospital of Stanford UniversityPalo Alto California 94304 United States
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Recruiting completed1Ann & Robert Lurie Children's Hospital of ChicagoChicago Illinois 60611 United States
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Recruiting completed1University of Illinois at Chicago Hospitals and Health SystemsChicago Illinois 60612 United States
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Recruiting completed1Columbia University Medical Center (21+ years)New York New York 10032 United States
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Recruiting completed1Columbia University Medical Center (≤21 years)New York New York 10032 United States
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Recruiting completed1Children's Hospital of PhiladelphiaPhiladelphia Pennsylvania 19104 United States
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Recruiting completed1St. Jude Children's Research HospitalMemphis Tennessee 38105 United States
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Recruiting completed1The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood CancersNashville Tennessee 37203 United States
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Recruiting completed1Methodist Children's Hospital/Texas Transplant InstituteSan Antonio Texas 78229 United States