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Lipodystrophy Connect Patient Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02577952
Recruitment Status : Completed
First Posted : October 16, 2015
Last Update Posted : April 22, 2019
Sponsor:
Information provided by (Responsible Party):
Vanessa Rangel Miller, PatientCrossroads

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

Condition or disease
Lipodystrophy

Detailed Description:
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.

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Study Type : Observational [Patient Registry]
Actual Enrollment : 257 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Target Follow-Up Duration: 5 Years
Official Title: Lipodystrophy Connect Patient Registry
Study Start Date : January 2014
Actual Primary Completion Date : November 8, 2018
Actual Study Completion Date : November 8, 2018

Resource links provided by the National Library of Medicine


Group/Cohort
People with Lipodystrophy & family
People currently living with lipodystrophy and their family members.



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
People with known or suspected lipodystrophy and their family members.
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02577952


Locations
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United States, California
PatientCrossroads
San Francisco, California, United States, 94103
Sponsors and Collaborators
PatientCrossroads
Investigators
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Principal Investigator: Vanessa Rangel Miller, M.S., M.B.A PatientCrossroads
Additional Information:
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Responsible Party: Vanessa Rangel Miller, Advocacy Partnerships, PatientCrossroads
ClinicalTrials.gov Identifier: NCT02577952    
Other Study ID Numbers: Pro00009092
First Posted: October 16, 2015    Key Record Dates
Last Update Posted: April 22, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: De-identified aggregate participant data is shared via a professional portal.
Keywords provided by Vanessa Rangel Miller, PatientCrossroads:
lipodystrophy
lipodystrophy, congenital generalized
Berardinelli-Seip Congenital Lipodystrophy
Congenital generalized lipodystrophy
generalized lipodystrophy
lipodystrophy, familial partial
partial lipodystrophy
lipodystrophy, partial, acquired
acquired lipodystrophy
Additional relevant MeSH terms:
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Lipodystrophy
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases