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Growth Hormone Receptor Antagonist

Pegvisomant for Growth Hormone Excess

Phase 3
Recruiting
Led By Christina Tatsi, M.D.
Research Sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Willingness to discontinue other medications for the treatment of GH excess for a 6-week washout period prior to initiating pegvisomant
Males and females 24 months to <18 years at enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial is testing the role of the drug pegvisomant in children and adolescents with gigantism. Eligible participants must be aged 2-18, have GH excess, and be unresponsive or ineligible for current treatments. The study will last 60 weeks and include several visits with medical history and physical exams, questionnaires, heart and liver tests, and blood and urine tests. Participants will also have an MRI and hand X-ray at the baseline and 12-month visits.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects of Pegvisomant may include reactions at the injection site like pain or swelling; changes in liver function; headaches; flu-like symptoms; nausea; potential interference with normal fat distribution.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am willing to stop my current GH excess treatments for 6 weeks before starting pegvisomant.
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I am between 2 and 17 years old.
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I have been diagnosed with excess growth hormone.
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My growth hormone-secreting tumor did not respond well to surgery or radiation.
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My growth hormone levels are higher than normal for my age and sex.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Determine the safety and tolerability of pegvisomant in children with GH excess
Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
Secondary outcome measures
Improvement in signs and symptoms of GH excess and quality of life from baseline to end of study (12 month visit)
Left ventricular ejection fraction change on echocardiogram from baseline to end of study (12 month visit).
Normalization of IGF-1 for age and sex from baseline to end of study (12 month visit)
+2 more

Side effects data

From 2017 Phase 4 trial • 44 Patients • NCT01701973
20%
Abdominal cramping and diarrhea
20%
Dizziness and paresthesias during arginine infusion
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Sitagliptin Plus Pegvisomant
Sitagliptin Plus LNMMA
Sitagliptin
Sitagliptin Plus Exendin 9-39

Trial Design

1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
Intervention arm, Patient received pegvisomant
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegvisomant
2010
Completed Phase 4
~320

Find a Location

Who is running the clinical trial?

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Lead Sponsor
1,955 Previous Clinical Trials
2,672,380 Total Patients Enrolled
Christina Tatsi, M.D.Principal InvestigatorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
1 Previous Clinical Trials
2,000 Total Patients Enrolled

Media Library

Pegvisomant (Growth Hormone Receptor Antagonist) Clinical Trial Eligibility Overview. Trial Name: NCT03882034 — Phase 3
Pituitary Disease Research Study Groups: 1
Pituitary Disease Clinical Trial 2023: Pegvisomant Highlights & Side Effects. Trial Name: NCT03882034 — Phase 3
Pegvisomant (Growth Hormone Receptor Antagonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03882034 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many test subjects are being approved for this experiment?

"That is accurate. The clinical trial is recruiting patients, as reflected on clinicaltrials.gov. This study was posted on October 21st, 2019, and was last updated on September 28th, 2020. They are seeking to enroll 140 individuals from 1 site."

Answered by AI

If a patient is not yet 55, can they still qualify for the research?

"The trial's inclusion criteria state that the youngest age a patient can be is 2 years old, with the maximum age being 18."

Answered by AI

Are there any health concerns that come with taking Pegvisomant?

"Pegvisomant has been determined to be a safe medication by our team, as it has received a score of 3. This score is given to Phase 3 drugs, which means that there is some data supporting efficacy and multiple rounds of data supporting safety."

Answered by AI

What other scientific research has been published about Pegvisomant?

"Currently, there are 2 ongoing Pegvisomant clinical trials. 1 of these active research studies is in Phase 3. Although the majority of Pegvisomant trials take place in Bethesda, Maryland, there are 2 clinical trial sites."

Answered by AI

Are participants being recruited for this trial at present?

"The trial is currently recruiting patients, as noted on clinicaltrials.gov. This page was originally created on October 21st, 2019 and was last updated on September 28th, 2020."

Answered by AI
~46 spots leftby Dec 2026