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

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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. 

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