120 Participants Needed

Pegvisomant for Growth Hormone Excess

CT
SM
SM
Overseen BySamah M Agabein, M.D.
Age: < 65
Sex: Any
Trial Phase: Phase 3
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Must be taking: Somatostatin analogues, Dopamine agonists
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

Research shows that Pegvisomant is effective in controlling acromegaly (a condition of excessive growth hormone) by normalizing disease activity and improving symptoms. It is well tolerated and has been shown to work when other treatments have failed.12345

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

CT

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

I am willing to stop my current GH excess treatments for 6 weeks before starting pegvisomant.
I am between 2 and 17 years old.
Able to provide consent/assent if developmentally appropriate
See 5 more

Exclusion Criteria

Positive pregnancy test in females, current pregnancy and/or female patients who are breastfeeding
Patients with any medical, physical, psychiatric, or social condition, which, in the opinion of the investigators, would make participation in this protocol not in their best interest
My liver tests are three times above the normal range.
See 1 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Baseline

Participants undergo baseline assessments including medical history, physical exam, and initial drug administration

1 week
1 visit (in-person, overnight stay)

Treatment

Participants receive daily injections of pegvisomant and undergo regular monitoring including monthly local doctor visits for height, weight, and blood tests

60 weeks
3 main visits (baseline, 6-month, 12-month), monthly local visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Pegvisomant
Trial Overview The trial tests Pegvisomant's safety and effectiveness in managing excessive growth hormone in kids. Over 60 weeks, participants will visit at least three times for medical exams, questionnaires, heart/liver tests, MRI scans with dye injection, hand X-rays and learn to self-inject the drug daily at home.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
Intervention arm, Patient received pegvisomant

Pegvisomant is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Somavert for:
  • Acromegaly
🇪🇺
Approved in European Union as Somavert for:
  • 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

Trials
2,103
Recruited
2,760,000+

Findings from Research

Pegvisomant is a growth hormone receptor antagonist that has shown to be highly effective in treating acromegaly, a condition caused by excess growth hormone.
Initial results indicate that pegvisomant is the most effective treatment for achieving biochemical control of the disease, as measured by levels of circulating IGF-I.
Metabolic effects of GH antagonism in patients with acromegaly.Trainer, PJ.[2019]
Pegvisomant, a growth hormone receptor antagonist introduced in 2004, provides an effective treatment option for patients with acromegaly who do not respond adequately to surgery, radiation, or other medical therapies, demonstrating good tolerance and safety based on the Acrostudy database.
Clinical trials are underway to explore the combination of pegvisomant with somatostatin analogs, which may enhance treatment efficacy and expand the potential uses of growth hormone receptor antagonists beyond acromegaly.
[Growth hormone receptor antagonist in the treatment of acromegaly].Hubina, E., Tóth, A., Kovács, GL., et al.[2016]
In the German Pegvisomant Observational Study (GPOS) involving 263 patients with acromegaly, pegvisomant treatment led to normalization of disease activity in most patients and improved glucose metabolism, demonstrating its efficacy as a growth hormone-receptor antagonist.
The safety profile of pegvisomant was favorable, with tumor growth rates similar to those seen with somatostatin analogues, and while some patients experienced transaminase elevations, these were mostly transient and resolved without long-term issues.
Experience from the German pegvisomant observational study.Strasburger, CJ., Buchfelder, M., Droste, M., et al.[2016]

References

Metabolic effects of GH antagonism in patients with acromegaly. [2019]
[Growth hormone receptor antagonist in the treatment of acromegaly]. [2016]
Experience from the German pegvisomant observational study. [2016]
Pegvisomant: current and potential novel therapeutic applications. [2021]
Pegvisomant: structure and function. [2021]
Cancer and the potential place for growth hormone receptor antagonist therapy. [2021]
Treatment of pituitary tumors: pegvisomant. [2018]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security