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About
This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.
Full description
CADPT03A12101 is a multicenter, multi-part, first-in-human, proof-of-concept, open label non-randomized, clinical study in Sickle Cell Disease (SCD) subjects. This study includes apheresis of mobilized hematopoietic stem and progenitor cells (HSPCs), ex vivo CRISPR/Cas9-mediated genome editing and expansion, followed by myeloablative conditioning and autologous hematopoietic stem cell transplant (HSCT) with follow-up for a minimum of one year and up to two years.
The study is divided into the following parts:
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Other protocol defined inclusion/exclusion criteria may apply
Primary purpose
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Interventional model
Masking
4 participants in 1 patient group
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Central trial contact
Novartis Pharmaceuticals; Novartis Pharmaceuticals
Data sourced from clinicaltrials.gov
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