Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
试验速览
- 阶段
- 1 期
- 状态
- Enrolling By Invitation
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Number of participants with adverse events -Safety by Incidence of Treatment-Emergent
研究概览
简要总结
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
详细描述
Patients who received EXG34217 treatment and had at least one follow-up visit (Month 1, 3, 6, or 12) in Study EXG-US-01 will be eligible for this rollover study. Patient will sign a consent form prior to any study related procedure. This study is to add additional follow-up assessments up to 6 years after EXG34217 treatment. This study does not have additional intervention. Additional visits will be every 6 months in the first 3 years and once a year for two 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
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures.
- •Have completed the 12-month visit of Study EXG-US-01.
排除标准
- 未提供
结局指标
主要结局
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent
时间窗: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Number of participants with a change in clinical laboratory evaluations
时间窗: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change in Electrocardiography (ECG)
时间窗: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
ECG (standard digital 12-lead in singlicate)
Number of participants with a change of Immunogenicity
时间窗: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Change in Antibody against virus vector and transgene
umber of participants with a change in in physical examination
时间窗: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
次要结局
- Number of participants with a change in telomere length(Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72)
- Number of participants with improvement of blood counts.(Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72)