Natural History of Pregnancy and Pregnancy Outcomes in Metreleptin-Treated vs Untreated Subjects With Lipodystrophy

NCT05419037 | Recruiting

• 2 other identifiers: 10000757000757-DK
• Study Type: observational
• Target: 90
• Locations: 1 country
• Sites: 1

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

Keywords

Metreleptin; Pregnancy; Off-Spring; Natural History

Outcome Measures

Primary Outcomes (1)

• type and frequency of pregnancy complications

  We will quantify a set of pregnancy and offspring outcomes and complications.

  at end of study

Study Arms (2)

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.

Eligibility Criteria

• Age: 6 Months - 98 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Women who were pregnant while taking metreleptin and their off-spring will be evaluated.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Lipodystrophy

Condition Hierarchy (Ancestors)

Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Rebecca J Brown, M.D.

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Megan S Startzell, R.N.

CONTACT

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

Rebecca J Brown, M.D.

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2022
• First Posted: June 15, 2022
• Study Start: September 7, 2022
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2029
• Last Updated: March 16, 2026

Record last verified: 2026-03-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)