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

A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease Identifier: NCT04443907

Novartis Reference Number: CADPT03A12101

Last Update: Sep 11, 2020

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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.

Sickle Cell Disease
Phase 1
Phase 2
Overall status 
Enrollment count 
20 participants
Start date 
Aug 25, 2020
Completion date 
Nov 20, 2023
2 Years - 40 Years (Child, Adult)


Single intravenous infusion of OTQ923 cell suspension
Single intravenous infusion of HIX763 cell suspension.
OTQ923 or HIX763
Single intravenous infusion of either OTQ923 or HIX763, based on review of data from Part A and/or Part B by Health agencies after a formal interim analysis

Eligibility Criteria

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

Study Locations

United States
Novartis Investigative Site
Memphis, 38105-3678
United States


Novartis Pharmaceuticals
Novartis Pharmaceuticals

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