Status:
TERMINATED
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
2-40 years
Phase:
PHASE1
Brief Summary
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 reduci...
Detailed Description
CADPT03A12101 was a multicenter, multi-part, first-in-human, proof-of-concept, open label non-randomized, clinical study in Sickle Cell Disease (SCD) subjects. This study included apheresis of mobiliz...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 25 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 6 2025
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT04443907
Start Date
August 25 2020
End Date
January 6 2025
Last Update
January 8 2026
Active Locations (3)
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1
University of Chicago
Chicago, Illinois, United States, 60637
2
Memorial Sloan Kettering Cancer Ctr
New York, New York, United States, 10065
3
St Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105-3678