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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
Must not have
Liver function abnormalities (ALT, AST) greater than or equal to 3 times ULN
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group
Pivotal Trial

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.
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.
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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My liver tests are three times above the normal range.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
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 study objectives
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%
Dizziness and paresthesias during arginine infusion
20%
Abdominal cramping and diarrhea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Sitagliptin Plus Pegvisomant
Sitagliptin Plus LNMMA
Sitagliptin
Sitagliptin Plus Exendin 9-39

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
2,055 Previous Clinical Trials
2,731,766 Total Patients Enrolled
Christina Tatsi, M.D.Principal InvestigatorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
5 Previous Clinical Trials
7,677 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
~35 spots leftby Dec 2026
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