Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance (NCT05470504) | Clinical Trial Compass
RecruitingPhase 2
Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance
United States25 participantsStarted 2023-01-23
Plain-language summary
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.
Who can participate
Age range
18 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* INCLUSION CRITERIA:
To be eligible to participate in this study, an individual must meet all the following criteria:
* Either
* Known pathogenic variant in the insulin receptor gene, either dominant negative or recessive, OR
* Clinical diagnosis of partial lipodystrophy based on reduction in adipose tissue outside the normal range in selected adipose depots (including, at a minimum, the gluteofemoral depot) with preservation of adipose tissue in other depots.
* Male or female, aged 18-70 years.
* Completed linear growth and puberty.
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this study:
* Use of niacin or other drugs that directly affect lipolysis within 8 weeks prior to enrollment.
* Patients taking anticoagulants (blood thinning medications).
* Use of non-steroidal anti-inflammatory medications (e.g., aspirin, ibuprofen) 2 weeks prior to the biopsy date (in patients who choose to undergo biopsy).
* Changes in medications for diabetes or dyslipidemia within 2 weeks prior to enrollment.
* Pregnancy or lactation.
* For females of reproductive potential: inability or unwillingness to use contraception during study participation and for an additional 1 month after the end of pegvisomant administration.
* For males of reproductive potential: inability or unwillingness to use condoms or other methods to ensure effective contraception with partner during the study and for an additional 1 month after t…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Glycerol rate of appearance (Ra) normalized to fat mass, Palmitate Ra normalized to fat mass
Timeframe: 1 month
Trial details
NCT IDNCT05470504
SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)