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).
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2 Years - 11 Years
Phase 3
Interventional
All
Biological
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 therapy failure or intolerance at any point in the past
Eligible for autologous stem cell transplant as per investigators judgment
Key
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.