Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients
概览
- 阶段
- 不适用
- 干预措施
- Long Term Follow-Up
- 疾病 / 适应症
- Menkes Disease
- 发起方
- Sentynl Therapeutics, Inc.
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Long Term Follow-Up on Survival
- 状态
- 进行中（未招募）
- 最后更新
- 2个月前
概览
简要总结
This study will collect long term follow-up data regarding overall survival and neurological parameters from patients previously identified and/or enrolled in Protocols 09-CH-0059 and 90-CH0149 and Historical Control data on Menkes disease patients.
详细描述
Primary Objective: 1. To provide additional clinical and safety data on long term following administration of Copper Histidinate treatment in patients treated with Copper Histidinate under Protocols 09-CH-0059 and 90-CH0149 and untreated patients previously identified and/or enrolled in Protocol 09-CH-0059. 2. To provide additional Historical Control data on Menkes disease patients.
研究者
入排标准
入选标准
- •The subject must have been previously identified and/or enrolled under Protocols 09-CH-0059 or 90-CH-0149; or An untreated Menkes disease patients for whom data collection is incomplete under Protocols 09-CH-0059 (Amendment M); or Other untreated Menkes disease patients.
- •Must sign and date an informed consent form by parent or legal guardian for this study prior to any assessment being done in this study. If the patient is \> 18 years of age, the patient must sign the informed consent.
- •Male or female, aged 0 to \< 65 years of age.
排除标准
- •\- Unwillingness/unable to participate in the study.
研究组 & 干预措施
Treated with Copper Histidinate
干预措施: Long Term Follow-Up
Historical Control
结局指标
主要结局
Long Term Follow-Up on Survival
时间窗: 12/01/2019 - 12/31/2022
The primary outcome measure will be overall survival.