Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- iECURE, Inc.
- 入组人数
- 13
- 试验地点
- 3
- 主要终点
- AE and SAE (incidence, severity, seriousness, and relatedness)
研究概览
简要总结
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
详细描述
This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 7 Months 至 15 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
- •Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
- •Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.
排除标准
- •1. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.
研究组 & 干预措施
Enrolled and Dosed
Previously dosed in a previous iECURE study.
干预措施: No Intervention (Other)
Enrolled but Not Dosed
Enrolled in a previous iECURE study, but not dosed.
干预措施: No Intervention (Other)
结局指标
主要结局
AE and SAE (incidence, severity, seriousness, and relatedness)
时间窗: Over 14.5 years post dosing with ECUR-506
Safety
Change from baseline over 14.5 years post infusion in length
时间窗: Over 14.5 years post dosing with ECUR-506
Length measured in cenitmeters
Change from baseline over 14.5 years post infusion in weight
时间窗: Over 14.5 years post dosing with ECUR-506
Weight measured in kilograms
Urinalysis (Dip Stick) Evaluations
时间窗: Over 14.5 years post dosing with ECUR-506
Urinalysis (Dip Stick) will be evaluated to monitor pathological changes to the participants urine and to monitor levels of Specific Gravity, pH, Glucose, Protein, Blood, Ketones, Bilirubin, Urobilinogen, Nitrite, Leukocyte esterase, red blood cell count, and white blood cell count as they relate to established local lab normal ranges.
次要结局
- qPCR measurement to evaluate the clearance of both vectors in blood over time.(Over 14.5 years post dosing with ECUR-506)
- qPCR measurement to evaluate the clearance of both vectors in saliva over time.(Over 14.5 years post dosing with ECUR-506)
- qPCR measurement to evaluate the clearance of both vectors in urine over time.(Over 14.5 years post dosing with ECUR-506)
- qPCR measurement to evaluate the clearance of both vectors in feces over time.(Over 14.5 years post dosing with ECUR-506)
- Percent Liver Transduction(Over 14.5 years post dosing with ECUR-506)
- Number of hyperammonemic crises (HAC)(Over 14.5 years post dosing with ECUR-506)
- Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event(Over 14.5 years post dosing with ECUR-506)
- Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.(Over 14.5 years post dosing with ECUR-506)
- Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.(Over 14.5 years post dosing with ECUR-506)
- Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis(Over 14.5 years post dosing with ECUR-506)
- Scavenger drug dose per body surface area (BSA)(Over 14.5 years post dosing with ECUR-506)
- Protein allowance g/kg(Over 14.5 years post dosing with ECUR-506)
- Concentration of blood urea nitrogen measurements(Over 14.5 years post dosing with ECUR-506)
- Time to liver transplant from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol)(Over 14.5 years post dosing with ECUR-506)
- Transplant free survival(Over 14.5 years post dosing with ECUR-506)
- Time to liver transplant or any cause of death from dosing to EOS.(Over 14.5 years post dosing with ECUR-506)
- Overall Survival: Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).(Over 14.5 years post dosing with ECUR-506)
- Time to any-cause death from dosing to EOS. Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).(Over 14.5 years post dosing with ECUR-506)