NCT03837483进行中（未招募）3 期
A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 试验地点
- 4
- 主要终点
- Annualized rate of moderate and severe bleeding episodes up to 1 year after gene therapy compared with 1 year before gene therapy
研究概览
简要总结
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: up to 65 years
- •Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
- •Severe Wiskott-Aldrich Syndrome (WAS) gene mutation, defined by literature data (genotype/phenotype studies).;
- •Absent WASP expression, assessed by flow cytometry;
- •Severe clinical score (Zhu clinical score ≥ 3);
- •No human leukocyte antigen (HLA)-identical related donor available for hematopoietic stem cells transplant (HSCT).
排除标准
- •End-organ dysfunction, severe active infection not responsive to treatment or other severe disease or clinical condition which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
- •Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome.
- •Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia , or other serious haematological disorders
- •Documented human immunodeficiency virus (HIV) infection
- •Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin
- •Symptomatic herpes zoster, not responsive to specific treatment
- •Evidence of acute tuberculosis
- •Acute or chronic stable Hepatitis B
- •Presence of positive Hepatitis C RNA test result at screening
- •Patients not eligible for mobilization protocols in order to obtain CD34+ cells
- •Previous Gene Therapy
结局指标
主要结局
Annualized rate of moderate and severe bleeding episodes up to 1 year after gene therapy compared with 1 year before gene therapy
时间窗: 12 months
Annualized rate of severe infections from 6 to 18 months after gene therapy compared with 1 year before gene therapy
时间窗: 18 months
次要结局
- The number of subjects presenting with malignancies or abnormal clonal proliferation(2 years)
- Percentage of WAS protein expression increased from pre-treatment levels in lymphocytes(2 years)
- Percentage of WAS protein expression increased from pre-treatment levels in platelets(2 years)
- Number of participants with successful engraftment of OTL-103(6 months)
- Number of patients with Vector copy number (VCN)/cell > 0.1 measured in peripheral blood-derived CD3+ cells(2 years)
- Evaluation of the overall survival(36 months)
研究者
研究点 (4)
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