RECRUITING

Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance

Conditions

Insulin Receptor Mutation Partial Lipodystrophy Pegvisomant Severe Insulin Resistance Growth Hormone Inhibition LIPOLYSIS

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Phase II Study of Growth Hormone Inhibition Using Pegvisomant In Severe Insulin Resistance

Quick Facts

Study Start:2023-01-23

Study Completion:2025-01-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05470504

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 18-65 years. * Completed linear growth and puberty.	* 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.

Inclusion Criteria

Exclusion 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 18-65 years.
* Completed linear growth and puberty.

* 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.

Contacts and Locations

Study Contact

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

Principal Investigator

Rebecca J Brown, M.D.

PRINCIPAL_INVESTIGATOR

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

Study Locations (Sites)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892

United States

Collaborators and Investigators

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

Rebecca J Brown, M.D., PRINCIPAL_INVESTIGATOR, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-01-23

Study Completion Date2025-01-30

Study Record Updates

Study Start Date2023-01-23

Study Completion Date2025-01-30

Terms related to this study

Keywords Provided by Researchers

Pegvisomant
Severe Insulin Resistance
Growth Hormone Inhibition
LIPOLYSIS

Additional Relevant MeSH Terms

Insulin Receptor Mutation
Partial Lipodystrophy