Lipodystrophy Connect Patient Registry
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 257
- 试验地点
- 1
- 主要终点
- Descriptive epidemiology of congenital and acquired lipodystrophy.
研究概览
简要总结
Lipodystrophy Connect is an online survey tool designed to collect demographic data and health information from individuals with Lipodystrophy.
详细描述
Lipodystrophy is a heterogeneous group of rare and inherited syndromes characterized by the complete or partial loss or absence of subcutaneous adipose tissue. People living with lipodystrophy lack the fat tissue required for normal metabolic function. To enhance knowledge of the underlying biology of these conditions and to institute effective clinical treatments, the Lipodystrophy community has developed a mechanism for individuals with any of the Lipodystrophy conditions to enroll in a Lipodystrophy registry called Lipodystrophy Connect. The objective of such a registry is to create a platform that connects all of the Lipodystrophy community, including persons with Lipodystrophy, family members, healthcare providers, researchers, drug companies, advocacy groups, federal research organizations, and regulators. A patient registry will provide the foundation for a resource to meet the current and future needs of the Lipodystrophy community as well as accelerate the rate of research and development of therapies that improve the quality of life of those with Lipodystrophy. Lipodystrophy Connect is an online survey tool that collects basic demographic information and health information from people with any rare form of Lipodystrophy.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Adults, > 18 years of age, with a diagnosis or suspected diagnosis of lipodystrophy, who are willing and able to provide informed consent, or for whom a legally authorized representative gives permission on behalf of the person to participate.
- •Minors (less than 18 years of age) with a diagnosis of lipodystrophy with a parent(s) or legally authorized representative who provides informed consent to participate and enter registry information on behalf of the minor. Note: Minors who are 7-17 years of age will be asked to provide assent to participate.
- •Adults > 18 years of age, without lipodystrophy, who are willing and able to provide informed consent and are family members of patients diagnosed with familial partial lipodystrophy (FPL) or congenital generalized lipodystrophy (CGL) and want to provide information about themselves.
排除标准
- •A person who does not have a suspected or confirmed diagnosis of Lipodystrophy.
- •A person who has a known diagnosis of HIV-associated Lipodystrophy.
结局指标
主要结局
Descriptive epidemiology of congenital and acquired lipodystrophy.
时间窗: Participants are requested to update their questionnaires every 12 months for 5 years.
Assessed from multiple questions regarding the natural history of patients with congenital or acquired lipodystrophy, with an emphasis on co-morbidities, history of medical evaluation or admission, and use of medications/alternative therapies.
次要结局
- Data on disease subtype(Participants are requested to update their questionnaires every 12 months for 5 years.)
- Change in congenital and acquired lipodystrophy clinical features(Participants are requested to update their questionnaires every 12 months for 5 years.)
- Change in patient self-report Quality of Life(Participants are requested to update their questionnaires every 12 months for 5 years.)
- Data on family and reproductive history(Participants are requested to update their questionnaires every 12 months for 5 years.)
研究者
Vanessa Rangel Miller
Advocacy Partnerships
PatientCrossroads