A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 66
- 试验地点
- 5
- 主要终点
- Evaluation of long-term safety of RGX-202
研究概览
简要总结
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.
详细描述
This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of investigational RGX-202. Eligible participants are those who have undergone evaluation in a previous (parent) clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD. Enrollment in the current long-term follow-up (LTFU) study will occur after the participant has completed or discontinued from the parent study. Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration (inclusive of the parent study). No investigational treatment will be administered under this protocol. The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •The parent(s) or legal guardian(s) of the participant has/(have) provided written informed consent and (where applicable) Health Insurance Portability and Accountability Act (HIPAA) authorization after the nature of the study has been explained, prior to any research-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
- •Must have undergone evaluation in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.
- •Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, and study procedures.
- •Sexually active participants must be willing to use a medically accepted method of contraception from the time of the screening visit through 5 years after RGX-202 administration.
排除标准
- •No exclusion criteria apply in this observational follow up study.
研究组 & 干预措施
RGX-202 Recipients
Subjects who have received RGX-202 in a separate parent study.
干预措施: No Intervention (Genetic)
结局指标
主要结局
Evaluation of long-term safety of RGX-202
时间窗: Baseline, 5 years inclusive of parent study
Incidences of AEs and SAEs over time
次要结局
- Time to Stand (TTSTAND)(Baseline, 5 years inclusive of parent study)
- Time to Run/Walk (TTRW)(Baseline, 5 years inclusive of parent study)
- Time to Climb (TTCLIMB)(Baseline, 5 years inclusive of parent study)
- North Start Ambulatory Assessment (NSAA)(Baseline, 5 years inclusive of parent study)
- Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Control Subtest(Baseline, 5 years inclusive of parent study)
- Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Transport Subtest(Baseline, 5 years inclusive of parent study)
- Stride velocity 95th centile (SV95C)(Baseline, 5 years inclusive of parent study)
- Pharmacokinetics (PK)(Baseline, 5 years inclusive of parent study)
- Vector Shedding(Baseline, 5 years inclusive of parent study)