Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Stopped early · Phase 1
Conditions studied: Sickle Cell Disease
In brief
This study evaluated 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.
Key facts
- Study ID
- NCT04443907
- Run by
- Novartis Pharmaceuticals
- People needed
- 4
- Starts
- 2020-08-25
- Expected to finish
- 2025-01-06
- Last updated by the study team
- 2026-01-08
Who can join
Age: 2 and older, up to 40. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- University of Chicago — Chicago, Illinois, United States
- Memorial Sloan Kettering Cancer Ctr — New York, New York, United States
- St Jude Children's Research Hospital — Memphis, Tennessee, United States
Full record on ClinicalTrials.gov
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