NCT02231697已完成不适用
The RECENSUS Study: A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)
Astellas Gene Therapies8 个研究点 分布在 2 个国家目标入组 161 人开始时间: 2014年9月最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 161
- 试验地点
- 8
- 主要终点
- Survival
研究概览
简要总结
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical manifestations and recorded medical management of XLMTM and potentially inform the design of future therapeutic intervention studies.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patient diagnosed with XLMTM resulting from a confirmed mutation in the MTM1 gene, or a combination of XLMTM genetically confirmed family history and muscle biopsy
- •Patient is male
- •Access to available medical records for each patient
- •Signed informed consent by the parent(s) or legal guardians and/or assent by the patient (when applicable), unless the associated IRB provides an appropriate consent waiver
排除标准
- •Patient data after participation in an interventional study designed to treat XLMTM (patient data prior to participation in an interventional study may be included)
结局指标
主要结局
Survival
时间窗: Lifetime, up to 50 years
Descriptive statistics: mean, median, range for age at death for deceased patients, mean, median, range for current age for living patients
次要结局
- Age at diagnosis(Lifetime, up to 50 years)
- Age at tracheostomy (if applicable)(Lifetime, up to 50 years)
- Age at need for/type of ventilation (if applicable)(Lifetime, up to 50 years)
研究者
研究点 (8)
Loading locations...
相似试验
已完成
不适用
Chart Review of Outcome of Treatment for S. Aureus BacteremiaStaphylococcus AureusNCT00304902Michael E. DeBakey VA Medical Center120
进行中（未招募）
不适用
A Multicenter, Retrospective, Chart Review Study Evaluating the Impact and Utility of the Blood-Based Proteomic Integrated Classifier and Auto-Antibody Tests in the Real-WorldLung NodulesNCT06728319Biodesix, Inc.4,000
已完成
不适用
A Retrospective Chart Review Study of the Outcomes of 2nd Line Therapy With LEn/Dex in Greek Patients With R/R Multiple MyEloma and the Treatment PatterNs Following Progressive DiseaseRelapsed/Refractory Multiple MyelomaNCT02608515Genesis Pharma S.A.207
已完成
不适用
A Retrospective Chart Review Study to Evaluate Safety of McGhan Single Lumen Gel-Filled Breast Implants in Patients in ChinaBreast AugmentationBreast ReconstructionNCT04261608Allergan225
已完成
不适用
A Study of Participants With Squamous Cell Oesophageal Carcinoma Receiving Nivolumab as Part of an Early Access to Medicines Scheme (EAMS) Program in the United KingdomEsophageal Squamous Cell CarcinomaNCT04936399Bristol-Myers Squibb87