Pegvisomant for Growth Hormone Excess
Trial Summary
Will I have to stop taking my current medications?
If you are taking medications for growth hormone excess, you can continue them as long as the dose is stable. If you want to stop those medications, you need a 6-week break before starting the trial.
What data supports the effectiveness of the drug Pegvisomant for growth hormone excess?
How is the drug Pegvisomant unique in treating growth hormone excess?
Pegvisomant is unique because it directly blocks the growth hormone receptor, preventing the hormone from working, which is different from other treatments that try to reduce hormone levels. It is particularly effective for patients who do not respond to other treatments and can normalize hormone levels in most patients.14567
What is the purpose of this trial?
Background:For children with gigantism, too much growth hormone (GH) in the body causes abnormal growth and many other problems. Current treatments often don t work; no medical treatment is approved by FDA. Researchers want to see if the drug pegvisomant can help.Objective:To test the role of pegvisomant in children and adolescents with gigantism.Eligibility:People ages 2-18 with GH excess for whom usual treatments have not worked or who are not eliginle for themDesign:Participants will be screened with a medical history.The study will last 60 weeks and include at least 3 visits: baseline, 6-month, and 12-month visits. For the baseline visit, participants will stay a few nights for testing. They may stay overnight for the other visits.All visits will include:Medical historyPhysical examQuestionnairesHeart and liver testsParticipants may be photographed in their underwear if they agree.Blood tests: Participants will get a catheter: A small plastic tube will be placed in an arm vein. For some tests, the blood may be drawn every 30 minutes over 3 hours. For other tests, blood will be drawn every 20 minutes over 12 hours. Only clinically necessary tests will be done in each patient.At the baseline visit, participants will have the study drug injected under the skin. They will learn to take the injection at home. They will take the injection daily during the study.The baseline and 12-month visits will include:MRI: Participants will have a dye injected into a vein. They will lie in a machine that takes pictures of the body.Hand X-rayParticipants must get their height and weight at their local doctor s office monthly.Participants must have blood and urine tests at their local lab monthly for the first 6 months then every 3 months until the study ends....
Research Team
Christina Tatsi, M.D.
Principal Investigator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Eligibility Criteria
This trial is for children and teens aged 2-18 with growth hormone excess, typically causing abnormal growth (gigantism), who haven't had success with standard treatments or can't use them. They must be willing to stop other GH treatments before starting the study drug, agree to certain birth control methods if applicable, have a local doctor for regular check-ups, and not have liver issues or current opioid use.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Baseline
Participants undergo baseline assessments including medical history, physical exam, and initial drug administration
Treatment
Participants receive daily injections of pegvisomant and undergo regular monitoring including monthly local doctor visits for height, weight, and blood tests
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Pegvisomant
Pegvisomant is already approved in United States, European Union for the following indications:
- Acromegaly
- Acromegaly
Find a Clinic Near You
Who Is Running the Clinical Trial?
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Lead Sponsor