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.
| Novartis Pharmaceuticals | |
| 1-888-669-6682 | |
| [email protected] |
| Novartis Pharmaceuticals | |
| +41613241111 | |
100 United States sites
25 Italy sites
2 Years - 40 Years
Phase 1/Phase 2
Interventional
All
Biological
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.
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