A first-in-patient Phase I/II clinical study to investigate the safety and efficacy of genome-edited hematopoietic stem and progenitor cells in subjects with severe complications of sickle cell disease - Study of safety and efficacy of genome-edited hematopoietic stem and progenitor cells in sickle cell

OVARTIS PHARMA AG0 个研究点目标入组 30 人开始时间: 2021年6月22日最近更新: 2024年3月12日

试验速览

暂无简介。

•Inclusion Criteria:
•1. Male or female subjects age 2-40 years inclusive
•2. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/ß0-thalassemia or others)
•3. Performance status >70% (Karnofsky for subjects >16 years of age and Lansky for subjects <16 years of age)
•4. 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
•5. Subjects, who have failed, not tolerated or refused hydroxyurea therapy.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 20
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Exclusion Criteria:
•1. Available matched related donor for HSCT
•2. Clinically significant active infection
•3. Seropositive for HIV or HTLV
•4. Active known malignancy, myelodysplasia, abnormal cytogenetics or
•immunodeficiency
•5. Prior HSCT or gene therapy
•6. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
•7. Protocol defined iron overload
•8. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya
•9. Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya
•Other protocol defined inclusion/exclusion criteria may apply

发起方