Recruiting

Metreleptin vs. Untreated

Sponsor:

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Code:

NCT05419037

Conditions

Lipodystrophy

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

Study Details

Brief 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. 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.

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....

Conditions

Lipodystrophy

Study ID

NCT05419037

Start date

Sep 7, 2022

Status verified date

Aug 14, 2026

Completion date

Mar 1, 2029

Anticipated

Primary completion date

Mar 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

  • INCLUSION CRITERIA:

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:

  • 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.

EXCLUSION CRITERIA:

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.

Study Design

Enrollment

90 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Primary outcome measure

  • type and frequency of pregnancy complications [ Time Frame: at end of study ]

Central Contacts and Locations

Central contacts

Megan S Startzell, R.N.

(301) 402-6371megan.startzell@nih.gov

Rebecca J Brown, M.D.

(301) 594-0609brownrebecca@mail.nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

800-411-1222ccopr@nih.gov

More Information

Sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Last update posted

Aug 18, 2026

Last verified

Aug 14, 2026

Keywords

  • Metreleptin
  • Pregnancy
  • Off-Spring
  • Natural History

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) on 2026-08-18.