Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

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Status and phase

Active, not recruiting
Phase 1


Sickle Cell Disease


Biological: OTQ923

Study type


Funder types



2019-003489-41 (EudraCT Number)

Details and patient eligibility


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: Part A - Adult subjects dosed with OTQ923. Part B - Assessment of OTQ923 in pediatric patients. Part B will not be opened.


5 patients




2 to 40 years old


No Healthy Volunteers

Inclusion criteria

  • Male or female subjects age 2-40 years inclusive
  • Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others)
  • Performance status >70% (Karnofsky for subjects >16 years of age and Lansky for subjects <16 years of age)
  • At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization
  • Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

Exclusion criteria

  • Available matched related donor for HSCT
  • Clinically significant active infection
  • Seropositive for HIV or HTLV
  • Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency
  • Prior HSCT or gene therapy
  • Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
  • Protocol defined iron overload
  • Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya
  • Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya

Other protocol defined inclusion/exclusion criteria may apply

Trial design

5 participants in 1 patient group

Experimental group
Single intravenous infusion of OTQ923 Part A - Adults treated with OTQ923; Part B - Children age 2-17 treated with OTQ923 based on review of data from Part A by Health agency after a formal interim analysis.
Biological: OTQ923
Biological: OTQ923

Trial contacts and locations



Central trial contact

Novartis Pharmaceuticals; Novartis Pharmaceuticals

Data sourced from

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