Lipodystrophy Connect Patient Registry

NCT02577952 | Completed

• 1 other identifier: Pro00009092
• Study Type: observational
• Target: 257
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Lipodystrophy

Keywords

lipodystrophy; lipodystrophy, congenital generalized; Berardinelli-Seip Congenital Lipodystrophy; Congenital generalized lipodystrophy; generalized lipodystrophy; lipodystrophy, familial partial; partial lipodystrophy; lipodystrophy, partial, acquired; acquired lipodystrophy

Outcome Measures

Primary Outcomes (1)

• Descriptive epidemiology of congenital and acquired lipodystrophy.

  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.

  Participants are requested to update their questionnaires every 12 months for 5 years.

Secondary Outcomes (4)

• 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.

Study Arms (1)

People with Lipodystrophy & family

People currently living with lipodystrophy and their family members.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

People with known or suspected lipodystrophy and their family members.

You may qualify if:

• 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.

You may not qualify if:

• A person who does not have a suspected or confirmed diagnosis of Lipodystrophy.
• A person who has a known diagnosis of HIV-associated Lipodystrophy.

Sponsors & Collaborators

• PatientCrossroads: lead

Study Sites (1)

PatientCrossroads

San Francisco, California, 94103, United States

Location

MeSH Terms

Conditions

Lipodystrophy; Lipodystrophy, Congenital Generalized; Lipodystrophy, Familial Partial; Lipodystrophy, Partial, Acquired

Condition Hierarchy (Ancestors)

Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Laminopathies

Study Officials

• Vanessa Rangel Miller, M.S., M.B.A

  PatientCrossroads

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Advocacy Partnerships

Study Record Dates

• First Submitted: October 8, 2015
• First Posted: October 16, 2015
• Study Start: January 1, 2014
• Primary Completion: November 8, 2018
• Study Completion: November 8, 2018
• Last Updated: April 22, 2019

Record last verified: 2019-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)