LipodystrophyNCT02577952
Lipodystrophy Connect Patient Registry
An observational study of Lipodystrophy, run by PatientCrossroads at 1 site in United States.
Trial summary
Lipodystrophy Connect is an online survey tool designed to collect demographic data and health information from individuals with Lipodystrophy. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Lipodystrophy
- How many people
- 257 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to 8 Nov 2018
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: People with known or suspected lipodystrophy and their family members.
Who may be able to join
- 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.
Who cannot join
- A person who does not have a suspected or confirmed diagnosis of Lipodystrophy.
- A person who has a known diagnosis of HIV-associated Lipodystrophy.
Trial locations and contacts
1 site in 1 country.
About this study
Lipodystrophy Connect is an online survey tool designed to collect demographic data and health information from individuals with Lipodystrophy.
Read the 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.
- 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
- Medical subject headings
- Lipodystrophy, Lipodystrophy, Congenital Generalized, Lipodystrophy, Familial Partial, Lipodystrophy, Partial, Acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| People with Lipodystrophy & family | People currently living with lipodystrophy and their family members. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Participants are requested to update their questionnaires every 12 months for 5 years.
Secondary outcomes (4)
Data on disease subtype
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.
Measured: Participants are requested to update their questionnaires every 12 months for 5 years.
Change in congenital and acquired lipodystrophy clinical features
Assessed from multiple questions regarding the common physical features hallmark to congenital and acquired lipodystrophy.
Measured: Participants are requested to update their questionnaires every 12 months for 5 years.
Change in patient self-report Quality of Life
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.
Measured: Participants are requested to update their questionnaires every 12 months for 5 years.
Data on family and reproductive history
Multiple questions regarding whether biological family members have been diagnosed with lipodystrophy or report lipodystrophy symptoms, and personal reproductive history.
Measured: Participants are requested to update their questionnaires every 12 months for 5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2015 (estimated)
- Study startJan 2014
- Primary completion8 Nov 2018
- Study completion8 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LipodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 257 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- PatientCrossroads
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Principal investigator, Vanessa Rangel Miller, PatientCrossroads
- Investigators
- Vanessa Rangel Miller, M.S., M.B.A, Principal investigator, PatientCrossroads
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02577952
- Sponsor's protocol ID
- Pro00009092
- Status last verified
- Apr 2019
- First submitted
- 8 Oct 2015
- Last updated
- 22 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: De-identified aggregate participant data is shared via a professional portal.
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lipodystrophy Connect Patient Registry
- ClinicalTrials.gov ID
- NCT02577952
- Acronym / sponsor ID
- Pro00009092
- Conditions
- Lipodystrophy
- Keywords
- lipodystrophy, lipodystrophy, congenital generalized, Berardinelli-Seip Congenital Lipodystrophy, Congenital generalized lipodystrophy, generalized lipodystrophy, lipodystrophy, familial partial, partial lipodystrophy, lipodystrophy, partial, acquired
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 257 participants (actual)
- Lead sponsor
- PatientCrossroadsIndustry (pharma, biotech, devices)
- Study start
- Jan 2014
- Primary completion
- 8 Nov 2018 (actual)
- Study completion
- 8 Nov 2018 (actual)
- First posted
- 16 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Lipodystrophy Connect is an online survey tool designed to collect demographic data and health information from individuals with 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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| People with Lipodystrophy & family | – | –People currently living with lipodystrophy and their family members. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- People with known or suspected lipodystrophy and their family members.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- A person who does not have a suspected or confirmed diagnosis of Lipodystrophy.
- A person who has a known diagnosis of HIV-associated Lipodystrophy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
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.
4 secondary outcomes
Data on disease subtype
Time frame: Participants are requested to update their questionnaires every 12 months for 5 years.
Change in congenital and acquired lipodystrophy clinical features
Time frame: Participants are requested to update their questionnaires every 12 months for 5 years.
Change in patient self-report Quality of Life
Time frame: Participants are requested to update their questionnaires every 12 months for 5 years.
Data on family and reproductive history
Time frame: Participants are requested to update their questionnaires every 12 months for 5 years.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2019.
- Individual participant dataSharing planned: No. De-identified aggregate participant data is shared via a professional portal.
Related trials for Lipodystrophy
Not yet recruitingPhase 2
Effects of Meal Macronutrients on Postprandial Lipids
Nephrotic Syndrome, Lipodystrophy
Active, not recruitingPhase 3
Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy
Lipodystrophy, Diabetes
Active, not recruitingPhase 2
Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
Diabetes, Lipodystrophy
RecruitingPhase 3
Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy
Generalized Lipodystrophy
Not yet recruitingObservational study
Sexual Quality of Life in Female Patients With Lipodystrophy Syndromes or Turner's Syndrome: a Case-control Study
Lipodystrophy Congenital Generalized, Lipodystrophy, Familial Partial
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vanessa Rangel Miller, M.S., M.B.A principal investigator
PatientCrossroads
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lipodystrophy, run by PatientCrossroads at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Descriptive epidemiology of congenital and acquired lipodystrophy. (time frame: Participants are requested to update their questionnaires every 12 months for 5 years.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2019.