Last updated on November 2019

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Blood disorder | SICKLE CELL ANEMIA | Hemoglobinopathy
  • Age: Between 18 - 35 Years
  • Gender: Male or Female

Key Inclusion Criteria:

  • Subjects 18 and 35 years of age
  • 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

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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