LipodystrophyNCT05419037
Natural History of Pregnancy and Pregnancy Outcomes in Metreleptin-Treated vs Untreated Subjects With Lipodystrophy
An observational study of Lipodystrophy, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.
Trial summary
Background: Lipodystrophy is a health problem in which the body does not have enough fat tissue. People with lipodystrophy may not make enough of the hormone leptin. Leptin regulates hunger. Low leptin levels trigger hunger. People with lipodystrophy can have many health problems. Read more about this study
- Who is it for?
- Ages 6 months to 98 years
- Conditions the study is for
- Lipodystrophy
- How many people
- 90 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Sep 2022to 1 Mar 2029
- Phase
- Observational
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 6 months to 98 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Women who were pregnant while taking metreleptin and their off-spring will be evaluated.
Who may be able to join
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Women with lipodystrophy who had pregnancies with or without use of metreleptin:
- Female, aged >= 18 years
- Clinical diagnosis of non-HIV associated generalized or partial lipodystrophy
- History of one or more pregnancies
- Offspring of women with lipodystrophy who had pregnancies while taking metreleptin:
Show all 10Show fewer
- Males or females aged >=1 month
- Mothers took metreleptin during their pregnancy
- Availability of a biobanked blood specimen or willingness to provide a blood specimen
- Note that subjects treated with metreleptin during pregnancy may participate in this study regardless of the participation of their offspring.
Who cannot join
- In order to be eligible to participate in this study, an individual must not meet any of the following criteria:
- Inability of subject or guardian to understand or the unwillingness to sign a written informed consent document (except as noted below with *)
- Pregnancy.
- Subjects who otherwise meet inclusion/exclusion criteria but who are not reachable to obtain informed consent may be included under a waiver of consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
About this study
Background:
Lipodystrophy is a health problem in which the body does not have enough fat tissue. People with lipodystrophy may not make enough of the hormone leptin. Leptin regulates hunger. Low leptin levels trigger hunger. People with lipodystrophy can have many health problems. They may take a drug (metreleptin) that mimics leptin. Little is known about how taking metreleptin may affect a pregnancy. Metreleptin may be helpful or harmful to pregnant women. It may also affect the health of the child who is born.
Read more
Objective:
This natural history study will collect data about the effects of taking metreleptin while pregnant.
Eligibility:
Women aged 18 years or older with lipodystrophy who have been pregnant. Women who did and who did not take metreleptin during their pregnancies are needed. Children of women with lipodystrophy who took this drug during pregnancy are also needed.
Design:
Participants will have 1 study visit. This visit may be by phone, by telehealth, or in-person.
Participants will answer questions about their pregnancies.
They will discuss any health problems they had.
They will be asked about any medicines they took before and during their pregnancies.
They will be asked about the health of their children.
Participants medical records will be reviewed.
Participants may need to provide a blood sample. They may also be asked to provide a sample of breastmilk.
Participants children may also be asked to provide a blood sample....
Read the detailed description
Study Description:
Single-site cross-sectional study of women with lipodystrophy who had pregnancies during which they were or were not exposed to metreleptin, and the offspring of women whose offspring were exposed to metreleptin during pregnancy. The study involves secondary use of data and samples from other studies in which these subjects participated, review of outside medical records, and collection of a single blood specimen if a biobanked specimen is not available.
The hypotheses are:
- Pregnancy complications will be less prevalent and severe in women with lipodystrophy who took metreleptin during pregnancy compared to those who did not, due the beneficial effects of metreleptin on metabolic complications of lipodystrophy.
- Anti-leptin antibodies may be detected in some offspring born to women with lipodystrophy treated with metreleptin during pregnancy, but these antibodies will not be associated with adverse health outcomes.
Objectives:
Primary: Determine pregnancy outcomes, including type and frequency of pregnancy complications in women treated with metreleptin during pregnancy vs. untreated pregnancies.
Secondary: Determine if anti-leptin antibodies are detectable in offspring of women treated with metreleptin during pregnancy.
Endpoints:
Primary Endpoint: We will quantify a set of pregnancy and offspring outcomes and complications.
Secondary Endpoints: Frequency and type of anti-leptin antibodies.
- Conditions
- Lipodystrophy
- Keywords
- Metreleptin, Pregnancy, Off-Spring, Natural History
- Medical subject headings
- Lipodystrophy
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 |
|---|---|---|
| off-spring of above | Off-spring of the women who took metreleptin during her pregnancy. | |
| Women with past pregnancy on metreleptin | Women who took metreleptin during pregnancy. |
What is being measured
The primary outcome is the main question the study is designed to answer.
type and frequency of pregnancy complications
We will quantify a set of pregnancy and offspring outcomes and complications.
Measured: at end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2022
- Study start7 Sep 2022
- Primary completion1 Mar 2028 (estimated)
- Study completion1 Mar 2029 (estimated)
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.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Rebecca J Brown, M.D., Principal investigator, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05419037
- Sponsor's protocol ID
- 10000757
- Other IDs
- 000757-DK
- Status last verified
- 14 Aug 2026
- First submitted
- 11 Jun 2022
- Last updated
- 18 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Result of the study will be disseminated to participants upon publication.