Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
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|ClinicalTrials.gov Identifier: NCT03882034|
Recruitment Status : Recruiting
First Posted : March 20, 2019
Last Update Posted : October 23, 2019
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.
To test the role of pegvisomant in children and adolescents with gigantism.
People ages 2-18 with GH excess for whom usual treatments have not worked or who are not eliginle for them
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:
Heart and liver tests
Participants 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.
Participants 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.
|Condition or disease||Intervention/treatment||Phase|
|Pituitary Disease||Drug: Pegvisomant||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Phase 3 Study of the Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess|
|Actual Study Start Date :||October 21, 2019|
|Estimated Primary Completion Date :||January 1, 2022|
|Estimated Study Completion Date :||January 22, 2022|
A fixed dose of pegvisomant 10-mg started on Day 1 of intervention and continued daily afterwards, will be administered subcutaneously according to manufacturer s recommendations. Thereafter, we will titrate the dosage to normalize serum IGF-1 concentrations as follows:-Increase the dosage by 5 mg increments every 4-12 weeks according to the study schedule of activities, if IGF-1 concentrations are greater than +2 SD for age and gender, to a maximum dose of 30mg per day.-Decrease the dosage by 5 mg decrements every 4-12 weeks according to the study schedule of activities, if IGF-1 concentrations are below -2 SD for age and gender, to a minimum dose of 5mg.
A fixed dose of pegvisomant 10-mg started on Day 1 of intervention and continued daily afterwards, will be administered subcutaneously according to manufacturer s recommendations. Thereafter, we will titrate the dosage to normalize serum IGF-1 concentrations as follows:
- Percent change of IGF-1 z-score from baseline to end of study (12 month visit). [ Time Frame: 1 year ]The primary endpoint is decrease in IGF-1 z-score >50% from baseline. This criterion will be used to determine efficacy.
- Determine the safety and tolerability of pegvisomant in children with GH excess [ Time Frame: During 1 year ]Safety will be determined by the periodical description of vital signs, laboratory and imaging studies, and other reported side effects.
- Normalization of IGF-1 for age and sex from baseline to end of study (12 month visit) [ Time Frame: 1 year ]
- Normalization of growth velocity [ Time Frame: 1 year ]
- Improvement in signs and symptoms of GH excess and quality of life from baseline to end of study (12 month visit) [ Time Frame: 1 year ]
- Left ventricular ejection fraction change on echocardiogram from baseline to end of study (12 month visit). [ Time Frame: 1 year ]
- Reduction of the left ventricular mass index (LVMi) on echocardiogram from baseline to end of study (12 month visit) [ Time Frame: 1 year ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03882034
|Contact: Christina Tatsi, M.D.||(301) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 email@example.com|
|Principal Investigator:||Constantine A Stratakis, M.D.||Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)|