Actively Recruiting
Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance
Led by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Updated on 2026-05-12
25
Participants Needed
1
Research Sites
261 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Lipodystrophy (LD) syndromes are a group of rare disorders that affect how a person s body can store and use fat tissue. Many people with LDs become severely insulin resistant. Some people are insulin resistant because of a variant in the insulin receptor gene. Insulin resistance causes many health problems. Objective: To learn if blocking the effects of growth hormone in the body will help people with severe insulin resistance. Eligibility: Adults aged 18 to 65 years with either a known variant in the insulin receptor gene or with a diagnosis of partial LD. Design: Participants will have 2 hospital stays, about 1 month apart. Each stay will be 3 or 4 nights. During each hospital stay, participants will have many tests. They will have a physical exam with blood tests. They will have all of their urine collected for a 24-hour period. They will have scans to measure their muscle, bone, and fat tissues. They will have tests to measure metabolism and insulin sensitivity. They may have an optional biopsy of fat tissue. During the first hospital visit, participants will learn how to give themselves shots of a drug (pegvisomant) that blocks growth hormone. The drug is injected under the skin. Participants will continue to give themselves these shots once a day at home. After the first hospital visit, participants will talk on the phone with members of the study team once each week. After 2 weeks they will have blood drawn for tests. Participants will stop the shots after the second hospital visit.
CONDITIONS
Official Title
Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 70 years
- Male or female participants who have completed puberty and linear growth
- Either have a known harmful variant in the insulin receptor gene OR a clinical diagnosis of partial lipodystrophy with specific fat tissue changes
You will not qualify if you...
- Use of niacin or other drugs affecting fat breakdown within 8 weeks before enrollment
- Taking blood thinning medications
- Use of non-steroidal anti-inflammatory drugs within 2 weeks before biopsy (if biopsy is chosen)
- Changes in diabetes or cholesterol medications within 2 weeks before enrollment
- Pregnancy or breastfeeding
- For women able to have children: unwillingness or inability to use contraception during study and 1 month after pegvisomant treatment
- For men able to father children: unwillingness or inability to use condoms or other contraception during study and 1 month after treatment
- Known allergy to pegvisomant or its ingredients
- Significant liver disease or abnormal liver tests
- Very high triglyceride levels at screening
- For partial lipodystrophy patients only: Hemoglobin A1c over 10% at screening
- Any other medical condition or medication that increases risk or affects study outcomes
- Inability or unwillingness to provide informed consent
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
M
Megan S Startzell, R.N.
CONTACT
R
Rebecca J Brown, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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