A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care.
研究概览
简要总结
To evaluate the clinical efficacy of FT007 infusion in terms of transfusion independence in pediatric and adult patients with transfusion dependent beta-thalassemia.
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Male and female adults/adolescents/children diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out of the 9 patients must have B0/B0 or B0/B0- like genotype. In case the genetic diagnosis available at screening wasn’t performed in a certified laboratory (check under PI’s or delegated investigator’s responsibility), the genetic diagnosis will be repeated at clinical sites during the screening phase
- •Documented history of at least 100 mL/kg/year or 10 U/year of packed red blood cell transfusions in each of the 2 years prior to signing informed consent.
- •Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group
- •Karnofsky Index or Lansky ≥ 80%
- •Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT
- •Low risk thrombophilic screen and negative history of significant previous thrombotic events
排除标准
- •Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long active agents)
- •Severe, active viral, bacterial or fungal infection at eligibility evaluation
- •Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
- •Current or prior immunodeficiency disorder.
- •For patients until the age of 14 years only: availability of an HLA-matched family donor
- •Previous allogeneic hematopoietic stem cell transplantation.
- •Previous gene therapy treatment (gene addition or gene editing).
结局指标
主要结局
Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care.
Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care.
次要结局
- Evaluation of the safety of treatment
- Overall survival at 12 and 24 months post GT.
- Evaluation of biological correlates of safety
研究者
Regulatory Affairs
Scientific
Fondazione Telethon Ets