💬In plain words
What the trial is, and whether it could suit you
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 the
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
* 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 the end of pegvisomant administration.
* Known allergic reactions pegvisomant or any of its components.
* Clinically significant liver disease, evidenced by any of the following:
* ALT or AST \>3 times the upper limit of normal at screening.
* Current known liver disease other than steatohepatitis (e.g., autoimmune or viral hepatitis).
* History of cirrhosis
* Triglycerides \>1500 mg/dL (non-fasting) or \>1000 mg/dL (fasting) at screening.
* In subjects with partial lipodystrophy only, Hemoglobin A1c \>10% at screening.
* Any other medical condition or medication that, in the judgement of the investigator, will increase risk to the subject or impede the measurement of study outcomes.
* Inability of subject to understand or the unwillingness to sign a written informed consent document.
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