Recruiting
Phase 3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

ClinicalTrials.gov Identifier

Eligibility criteria
Sex
Age
2 Years - 11 Years
Phase
3
Key inclusion criteria
  • Diagnosis of severe SCD as defined by:
  • Documented SCD genotypes
  • History of at least two severe VOCs events per year for the previous two years prior to enrollment
  • Hydroxyurea (HU) failure unless HU intolerant
  • Eligible for autologous stem cell transplant as per investigators judgment
Key exclusion criteria
  • A willing and healthy 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, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
Study duration
May 2022 - May 2026
Study type
Interventional
Intervention/Treatment
Biological : CTX001
Estimated enrollment
15

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Location
  • Recruiting
    1
    Children's Hospital of Philadelphia
    Philadelphia Pennsylvania 19104 United States
  • Recruiting
    1
    St. Jude Children's Research Hospital
    Memphis Tennessee 38105 United States
  • Recruiting
    1
    The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
    Nashville Tennessee 37203 United States