A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This is a multinational, long-term follow-up study to assess the long-term safety and durability of CTNS-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy. No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from the initial date of CTNS-RD-04 infusion.
详细描述
Participants enrolled in a study where the individual received CTNS-RD-04 will be offered participation in the CTNS-RD-04-LTF01 study. The Baseline visit for the CTNS-RD-04-LTF01 study will likely coincide with the final visit in the parent study. Participants confirmed eligible for the CTNS-RD-04-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for up to 11 years until a total of 15 years have elapsed during which time continued safety, engraftment, and efficacy of CTNS-RD-04 treatment will be assessed.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 14 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must have received CTNS-RD-04 in a preceding study
排除标准
- •Participant is currently enrolled in an CTNS-RD-04 treatment study. Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.
结局指标
主要结局
Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline to Year 15 post gene therapy
Number of participants with clinically relevant abnormalities, as assessed by by electrocardiograms (ECGs) (rate, rhythm, intervals)
时间窗: Baseline to Year 15 post gene therapy
Number of participants with clinically relevant abnormalities, as assessed by vital sign (heart rate, pulse rate, and temperature)
时间窗: Baseline to Year 15 post gene therapy
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests (chemistry and hematology)
时间窗: Baseline to Year 15 post gene therapy
次要结局
- Change from baseline in Cystinosin (CTNS) as assessed by quantitative Polymerase Chain Reaction (qPCR)(Baseline to Year 15 post gene therapy)
- Change from baseline in Cystine levels in leukocytes measured by mass spectrometry(Baseline to Year 15 post gene therapy)
- Change from baseline in ovarian reserve and menstrual cycle as assessed by anti-Müllerian hormone (AMH) and gynecology exams(Baseline to Year 15 post gene therapy)
- Change in male reproductive potential as assessed by urology exams (sperm count, motility, and morphology)(Baseline to Year 15 post gene therapy)
- Change from baseline in Renal glomerular and tubular functions measured by glomerular filtration rate (GFR)(Baseline to Year 15 post gene therapy)
- Change from baseline in respiratory function measured by spirometry(Baseline to Year 15 post gene therapy)
- Change from baseline in neurological function assessed by neurological exam (mental status, coordination, sensory, reflexes, and visual motor integration)(Baseline to Year 15 post gene therapy)
- Change from baseline in mean Vector Copy Number (VCN) as assessed by quantitative Polymerase Chain Reaction (qPCR)(Baseline to Year 15 post gene therapy)
- Change from baseline in Corneal cystine crystal score (CCCS) as assessed by in vivo confocal microscopy (IVCM)(Baseline to Year 15 post gene therapy)
- Change from baseline in vision function as assessed by ophthalmology exams(Baseline to Year 15 post gene therapy)
- Change from baseline in Endocrine function measured by fasting glucose, thyroid function, and gonadotropin levels(Baseline to Year 15 post gene therapy)
- Change from baseline in grip strength measured by dynamometry(Baseline to Year 15 post gene therapy)
- Change from baseline in bone density assessed by dual-energy X-ray absorptiometry (DEXA)(Baseline to Year 15 post gene therapy)
- Absence of Replication Competent Lentivirus (RCL) as assessed by Elisa assay(Baseline to Year 15 post gene therapy)
- Change from baseline in muscle mass assessed by dual-energy X-ray absorptiometry (DEXA)(Baseline to Year 15 post gene therapy)
- Change from baseline in psychometric function assessed by neurological exam (memory, oromotor function, intelligence quotient (IQ))(Baseline to Year 15 post gene therapy)
- Change from baseline in pill/injection count related to cystinosis treatment(Baseline to Year 15 post gene therapy)
研究者
Stephanie Cherqui
Principal Investigator
University of California, San Diego