CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 240
- 试验地点
- 10
- 主要终点
- Diagnosis
研究概览
简要总结
CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).
详细描述
Individuals can participate through the CureDuchenne Link™ application (accessible via mobile device or web interface) and receive communications about research opportunities and community programs. Participation may be done using virtual methods, at a project site, and/or at community events nationwide.
All collected information will be stored in a secure, HIPAA-compliant data warehouse for approved researchers to use for studies relevant to DMD, BMD and other neuromuscular disorders. Combining health and outcomes data with biospecimens provides an impactful solution and novel resource for researchers, allowing for effective translational research.
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
- 4 Weeks 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Any of the following are true:
- •Currently has a confirmed diagnosis of DMD/BMD based on genetic testing, muscle biopsy, or clinical diagnosis.
- •Currently has a confirmed diagnosis of carrier status for DMD/BMD based on genetic testing.
- •Parent/guardian (for minor participants) or participant gives informed consent and/or assent as required by local regulations.
- •Is age 4 weeks or older at the time of consent.
排除标准
- •Is a foster child or ward of the state.
- •Is a prisoner.
研究组 & 干预措施
Duchenne and Becker muscular dystrophy
Individuals with Duchenne muscular dystrophy and Becker muscular dystrophy
Carriers
Carriers of Duchenne muscular dystrophy and Becker muscular dystrophy
结局指标
主要结局
Diagnosis
时间窗: Upon study entry
There is no intervention in this project. Participants will provide documentation to support their diagnosis of Duchenne muscular dystrophy, Becker muscular dystrophy, or a carrier of these mutations
Genetic Mutation
时间窗: Upon study entry or when genetic testing results are available
Participants will be asked to provide genetic testing reports confirming their diagnosis, where available, which will be reviewed by a central genetic counselor.
次要结局
- Cardiac Status(Upon study entry and every 6-12 months thereafter for up to ten (10) years)
- Corticosteroid Status(Upon study entry and every 6-12 months thereafter for up to ten (10) years)
- Functional Status(Upon study entry and every 6-12 months thereafter for up to ten (10) years)
- North Star Ambulation Assessment (NSAA) Score(Upon study entry and every 6-12 months thereafter for up to ten (10) years)
- 6 Minute Walk Test (6MWT) Score(Upon study entry and every 6-12 months thereafter for up to ten (10) years)
- Respiratory Status(Upon study entry and every 6-12 months thereafter for up to ten (10) years)