A Long-term Safety and Efficacy follow-on Study in Participants With Transfusion Dependent Beta-thalassemia Who Have Previously Received OTL-300 (Formerly Know as GSK2696277)) and Completed the TIGET-BTHAL Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Number of subjects with absence of abnormal clonal proliferation (ACP)
研究概览
简要总结
OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
盲法说明
No study treatment will be administered.
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with OTL-
- •For adults; capable of giving signed informed consent. For children; informed assent and/or consent in writing signed by the subject and/or parent(s) / legal representative (according to local regulations and age of the subject).
排除标准
- 未提供
结局指标
主要结局
Number of subjects with absence of abnormal clonal proliferation (ACP)
时间窗: Up to 6 years
Clonal proliferation describes the selection and reproduction of only one type of cell.
Number of subjects with Polyclonal engraftment
时间窗: Up to 6 years
Integration site analysis will be performed on different hematopoietic lineages from peripheral blood and/or bone marrow. Polyclonality of hematopoiesis is defined as \>1000 unique integration sites retrieved at specified time points. The number of subjects with polyclonality of hematopoiesis will be estimated.
次要结局
- Number of subjects with adverse events (AEs), serious AEs (SAEs)(Up to 6 years)
- Number of subjects with reduction in red blood cells (RBC) transfusion volume(Up to 6 years)
- Number of subjects with reduction in transfusion rate up to transfusion independence(Up to 6 years)
- Number of subjects with transfusion independence(Up to 6 years)
- Hemoglobin (Hb) levels in subjects achieving transfusion independence(Up to 6 years)
- Number of subjects with sustained engraftment of genetically corrected cells(Up to 6 years)
- Number of subjects with overall survival(Up to 6 years)
- Clinical chemistry laboratory parameters as a measure of safety(Up to 6 years)
- Hematology laboratory parameters as a measure of safety(Up to 6 years)
- Urinalysis as a measure of safety(Up to 6 years)
- Occurrence of viral infections as a measure of safety(Up to 6 years)
- Screening for occurrence of antibodies against viruses and toxoplasma as a measure of safety(Up to 6 years)
- Functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scores(Up to 6 years)
- Short-Form-36 (SF-36) scores(Up to 6 years)
- Pediatric Quality of Life (PedsQL) questionnaire scores(Up to 6 years)
- Evaluation of growth in pediatric subjects(Up to 6 years)
- Assessment of hormonal levels in pediatric subjects(Up to 6 years)
- Changes in puberty status as assessed by clinical examination(Up to 6 years)
- Changes in puberty status as assessed by Tanner scale (TS)(Up to 6 years)
- Changes in puberty status as assessed by general questioning(Up to 6 years)