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

  • End date
    Aug 19, 2025
  • participants needed
  • sponsor
    Novartis Pharmaceuticals
Updated on 1 July 2022
acute chest syndrome
chest syndrome
sickle hemoglobin
hemoglobin f
hemoglobin c disease


This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Condition Sickle Cell Disease
Treatment OTQ923, HIX763, OTQ923 or HIX763
Clinical Study IdentifierNCT04443907
SponsorNovartis Pharmaceuticals
Last Modified on1 July 2022


Yes No Not Sure

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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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