Clinical Trial: Lipodystrophy Connect Patient Registry

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Lipodystrophy Connect Patient Registry

Brief Summary: Lipodystrophy Connect is an online survey tool designed to collect demographic data and health information from individuals with Lipodystrophy.

Detailed Summary: 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.
Sponsor: PatientCrossroads

Current Primary Outcome: Descriptive epidemiology of congenital and acquired lipodystrophy. [ Time Frame: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Data on disease subtype [ Time Frame: Participants are requested to update their questionnaires every 12 months for 5 years. ]
    Multiple questions about the patient's diagnostic process including reported diagnosis and subtype, time to diagnosis, method of diagnosis, age at diagnosis, presenting symptoms and specialty of diagnosing clinician.
  • Change in congenital and acquired lipodystrophy clinical features [ Time Frame: Participants are requested to update their questionnaires every 12 months for 5 years. ]
    Assessed from multiple questions regarding the common physical features hallmark to congenital and acquired lipodystrophy.
  • Change in patient self-report Quality of Life [ Time Frame: Participants are requested to update their questionnaires every 12 months for 5 years. ]
    Assessed from self-report quality-of-life (QoL) questions about the patient's overall health, everyday living, recent impact, personal impact, available resources, diet and lifestyle history.
  • Data on family and reproductive history [ Time Frame: Participants are requested to update their questionnaires every 12 months for 5 years. ]
    Multiple questions regarding whether biological family members have been diagnosed with lipodystrophy or report lipodystrophy symptoms, and personal reproductive history.


Original Secondary Outcome: Same as current

Information By: PatientCrossroads

Dates:
Date Received: October 8, 2015
Date Started: January 2014
Date Completion: December 2019
Last Updated: November 7, 2016
Last Verified: November 2016