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New treatment option for Insulin Receptor Mutation
Official title Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance
ClinicalTrials.gov ID: NCT05470504
What this study is testing
What is Pegvisomant?
Pegvisomant is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for insulin receptor mutation.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70
You may be able to join if
- 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...
- Male or female, aged 18-70 years.
You likely can't join if
- 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...
- Changes in medications for diabetes or dyslipidemia within 2 weeks prior to enrollment.
- Pregnancy or lactation.
See the full eligibility 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...
- Male or female, aged 18-70 years.
- Completed linear growth and puberty.
- 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...
- 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 people 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.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.