Pituitary DiseasePhase 3NCT03882034
Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
A Phase 3 study testing Pegvisomant in people with Pituitary Disease, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Pituitary Disease
- What is being tested?
- Pegvisomant
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Oct 2019to 30 Sep 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who are eligible for enrollment must meet the following eligibility criteria:
- Cohort 1: Patients with GH excess
- Males and females 24 months to <18 years at enrollment
- Active GH excess as demonstrated by the following:
- IGF-1 greater than the upper limit of normal for age and sex during screening (>+2 SD) and
- Abnormal GH levels as demonstrated by inability to suppress to <1 ng/mLwith older radioimmunoassays or <0.4ng/ml with sensitive immunoradiometric or immunochemiluminescent assays within 2 hours during Oral Glucose Tolerance Test (OGTT) after the administration of 1.75gr/kg (max 75gr) of glucose or elevated GH secretion profile during overnight sampling.
Show all 16Show fewer
- History of inadequate response to trans-sphenoidal surgery or radiation therapy for GH secreting pituitary tumor, or inability to tolerate surgery or radiation therapies or patient deemed inappropriate candidate for surgery and/or pituitary radiation therapy, as determined by review of the medical records by the PI. The evaluation of the patient should be performed at 3 months after the surgery date in order to ensure that there is persistent GH excess after the transsphenoidal resection of the tumor unless there are clear evidence of persistent disease, e.g. residual tumor, based on the PI s assessment. If the patient has received irradiation, there is no minimum time to be considered before enrolling in the study. The effects of radiation therapy take place over many years after receiving it (mean time to remission for stereotactic radiation therapy of 12-60 months), and, thus, a medical therapy is required during that period.
- Patient is either not receiving any other medication for GH excess or is receiving stable dose/frequency of other medications for the treatment of GH excess prior to initiation of pegvisomant and no further adjustment or addition of new medication is made for the duration of the study.
- Medications that may be co-administered for GH excess include but are not limited to: IM/SC/oral first- or second-generation somatostatin analogues (octreotide, lanreotide, pasireotide), oral dopamine agonist (cabergoline) and any other medication/formulation that may become available during this study. For each medication, its specific formulation and route of administration, patient will need to be on stable dose/frequency for a duration that is at least the minimum time recommended for adjusting the dose by the manufacturer which usually incorporates the time needed to reach plasma steady-state levels and a new plateau on the effect of the medication on IGF-1 (cabergoline: 4 weeks; SC octreotide/pasireotide administered twice or three times daily: 2 weeks; oral octreotide: 2 weeks; depot IM octreotide/SC lanreotide/IM pasireotide administered Q4 weeks: 3 months). The duration needed for the stable dose for patients on alternative schedules of administration of the above or other medications will be assessed based on available information and the longest estimated interval to achieve the full effect.
- If a patient has been receiving a medication for GH excess but is not interested to continue that medication a minimum period of 6-weeks of discontinuation of the medication will be required for washout. This period was defined in the prior version of the protocol and is designed to minimize the time the patient may not be receiving any treatment for GH excess.
- Able to provide consent/assent if developmentally appropriate
- Willing to use non-hormonal method of contraception in patients of reproductive potential from the start of the study until at least 28 days after they stop the medication. Females of reproductive age (Tanner 3 or more, and/or having menstrual cycle) will be educated on the risks of unknown potential fetal harm while using the investigational medication, and they will be educated on the alternative preventative methods for contraception (condoms). Females already receiving oral contraceptive pills (OCPs) will be evaluated by gynecology consult service to discuss effective non-hormonal contraception. Sexually active female subjects must agree to use an effective non-hormonal contraception for the duration of the study.
- Have a primary health care provider in home location who will perform regular height and weight measurements, vital signs, and safety labs.
- Height and weight will be requested to be performed according to the published methods included in the CDC-NHANES manual on anthropometry procedures manual (Supplementary Material). They will be plotted on the respective growth charts produced by the CDC for the US population (Supplementary Material).
- Cohort 2: Parents
- -Biological parents of a participant enrolled in the study enrolled for anthropometric measurements only.
Who cannot join
- Cohort 1: Patients
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Liver function abnormalities (ALT, AST) greater than or equal to 3 x ULN
- Positive pregnancy test in females, current pregnancy and/or female patients who are breastfeeding.
- Patients currently using opioids. Opioids induce altered metabolism of pegvisomant. Since this is a phase 3 study, opioids may affect the PK studies to be performed and, thus, chronic use of opioids (>2 weeks) will be an exclusion criterion.
- 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, will be excluded from the study. Patients who are critically ill, unstable, or with severe organ failure that may affect/limit the endocrine evaluation and place unsustainable demands on CC or NICHD resources will be excluded.
- Cohort 2: Patients Parents:
- Subjects enrolled as parents of patients do not have to be tested for the above exclusion criteria as their participation is limited to anthropometric measurements (no risk).
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: An Open-Label Phase 3 Study of the Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
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.
Read more
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 them
Design:
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 history
Physical exam
Questionnaires
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.
Hand X-ray
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.
...
Read the detailed description
Study Description:
GH excess is a rare and potentially lethal condition associated with hypersecretion of GH, usually by a pituitary tumor or hyperplasia. When it occurs prior to the complete fusion of growth plates, it leads to pathological tall stature, and it is called gigantism. After the fusion of the growth plates, it is called acromegaly. It may be associated with debilitating cardiovascular disease and/or diabetes. Children and adolescents with gigantism are currently treated with surgery, radiation therapy, and medications, such as octreotide, to reduce hypersecretion of GH; however, these treatments may lack efficacy and have significant side effects. Pegvisomant is a genetically engineered GH-receptor (GHR) antagonist that blocks the action of GH. In adults with acromegaly, pegvisomant has been shown to effectively reduce serum insulin-like growth factor type 1 (IGF-1) concentrations and lead to clinical improvement. However, experience in children and adolescents is limited to a small number of case series.,We propose the initiation of a new protocol at the NICHD, NIH, to treat children and adolescents with GH excess that is refractory to surgical therapy and/or radiation therapy, or in children and adolescents where the above therapies are contraindicated.
Objectives:
PRIMARY OUTCOMES:
- Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
- Determine the safety and tolerability of pegvisomant in children and adolescents with GH excess.
SECONDARY OUTCOMES:
- Percent change of IGF-1 z-score from baseline to end of the study in subgroups of patients receiving monotherapy and patients receiving additional medication that target GH excess.
- Normalization of the IGF-1 for age and sex from baseline to end of study (normal value defined as +/- 2 SD from the mean). Change in growth velocity to a near-normal range (+/- 1 SD) according to the Tanner and Davies growth velocity curves for age, sex, and stage of puberty, when comparing the 6-month period prior to the study drug initiation to the growth velocity between 6 and 12 months on the study drug.
- Improvement in signs and symptoms of GH excess that are common in the pediatric population (headaches, excessive perspiration, fatigue, increased appetite) and the quality of life of the patients from baseline to the end of the study (12 month visit).
- Improvement of the cardiac structure and function: reduction of the left ventricular mass index (LVMi), and change of the left ventricular ejection fraction (EF) on echocardiogram from baseline to the end of the study (12 month visit).
The objectives of the proposed study are to characterize the efficacy of pegvisomant as indicated by adequate control of the IGF-1 levels, the safety profile of the medication in children with GH excess, and to obtain pharmacodynamic data on the effect of pegvisomant on the GH-IGF axis in children. Because pegvisomant does not inhibit GH secretion, serum IGF-1 is the best marker of treatment efficacy. Pharmacokinetic (PK) studies will be performed in a subset of patients. This study will enroll patients with GH excess younger than 18 years and their biological parents.
Endpoints: Safety
Patients who have received at least 1 dose of pegvisomant will be included in the safety evaluations.
Safety will be evaluated by:
- Spontaneously reported adverse events
- Vital signs and periodic physical examinations (performed at the Clinical Center at baseline, 6 months (if possible unless telehealth visit performed) and 12 months after the study enrollment, and at local provider s office at regular intervals between those visits)
- Laboratory tests (performed periodically as indicated by previous studies) including:
- Hematology
- Chemistry
- Lipids
- Liver function panel
- Thyroid function tests
- IGF-1
- Glucose and insulin
- MRI of the pituitary to check for tumor growth at baseline, 6 months, and 1 year
- Annual liver ultrasound
- ECG and echocardiogram at baseline, 6 months (EKG, if possible unless telehealth visit performed) and 1-year to monitor for the presence of cardiac arrhythmias and GH related cardiomyopathy
- Care providers will complete the Gigantism Symptoms Assessment Questionnaire (GSAQ) and Child Health Questionnaire by Landgraf \& Wade for assessment of quality of life.
- Patients with gigantism and diabetes mellitus may require careful monitoring and dose reductions of insulin and/or oral hypoglycemic agents as pegvisomant usually has beneficial effects on glucose metabolism.7 Patients with pre-existing diabetes will continue to monitor their daily blood glucose levels, as previously instructed, and communicate with the investigational team for persistent elevation or decrease of the glucose levels. The changes of the dose of the anti-diabetic drugs and/or insulin will be done with communication of the research team with the local endocrinologist. Patients without pre-existing diabetes will be educated on the signs of hyperglycemia and will get monthly fasting blood glucose level measurements for the first 6 months along with the screening of the liver enzyme levels.
- Conditions
- Pituitary Disease
- Being tested
- Pegvisomant
- Keywords
- Pediatric, Gigantism, Growth Hormone, Hormone, Pituitary Disease
- Medical subject headings
- Pituitary Diseases, Gigantism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegvisomantDrug
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. Adjustment of the dose will occur as per protocol.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Intervention arm, Patient received pegvisomant Receives: Drug: Pegvisomant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
The primary endpoint is decrease in IGF-1 z-score >50% from baseline. This criterion will be used to determine efficacy.
Measured: 1 year
Determine the safety and tolerability of pegvisomant in children with GH excess
Safety will be determined by the periodical description of vital signs, laboratory and imaging studies, and other reported side effects.
Measured: During 1 year
Secondary outcomes (5)
Normalization of IGF-1 for age and sex from baseline to end of study (12 month visit)
Normalization of the IGF-1 for age and sex from baseline to end of study (normal value defined as +/- 2 SD from the mean).
Measured: 1 year
Normalization of growth velocity
Change in growth velocity to a near-normal range (+/- 1 SD) according to the Tanner and Davies growth velocity curves for age, sex, and stage of puberty, when comparing the 6-month period prior to the study drug initiation to the growth velocity between 6 and 12 months on the study drug.
Measured: 1 year
Improvement in signs and symptoms of GH excess and quality of life from baseline to end of study (12 month visit)
Improvement in signs and symptoms of GH excess that are common in the pediatric population (headaches, excessive perspiration, fatigue, increased appetite) and the quality of life of the patients from baseline to the end of the study (12 month visit).
Measured: 1 year
Left ventricular ejection fraction change on echocardiogram from baseline to end of study (12 month visit).
Improvement of the cardiac structure and function: reduction of the left ventricular mass index (LVMi), and change of the left ventricular ejection fraction (EF) on echocardiogram from baseline to the end of the study (12 month visit).
Measured: 1 year
Reduction of the left ventricular mass index (LVMi) on echocardiogram from baseline to end of study (12 month visit)
(LVMi), and change of the left ventricular ejection fraction (EF) on echocardiogram from baseline to the end of the study (12 month visit).
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2019
- Study start21 Oct 2019
- Primary completion8 Sep 2025
- Study completion30 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Pituitary DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Karim A Calis, Pharm.D., Principal investigator, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03882034
- Sponsor's protocol ID
- 190071
- Other IDs
- 19-CH-0071
- Status last verified
- 4 Nov 2025
- First submitted
- 19 Mar 2019
- Last updated
- 6 Nov 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Protocol is silent on IPD sharing.
Publications
Trainer PJ, Drake WM, Katznelson L, Freda PU, Herman-Bonert V, van der Lely AJ, Dimaraki EV, Stewart PM, Friend KE, Vance ML, Besser GM, Scarlett JA, Thorner MO, Parkinson C, Klibanski A, Powell JS, Barkan AL, Sheppard MC, Malsonado M, Rose DR, Clemmons DR, Johannsson G, Bengtsson BA, Stavrou S, Kleinberg DL, Cook DM, Phillips LS, Bidlingmaier M, Strasburger CJ, Hackett S, Zib K, Bennett WF, Davis RJ. Treatment of acromegaly with the growth hormone-receptor antagonist pegvisomant. N Engl J Med. 2000 Apr 20;342(16):1171-7. doi: 10.1056/NEJM200004203421604.
PubMed 10770982Lodish MB, Trivellin G, Stratakis CA. Pituitary gigantism: update on molecular biology and management. Curr Opin Endocrinol Diabetes Obes. 2016 Feb;23(1):72-80. doi: 10.1097/MED.0000000000000212.
PubMed 26574647Katznelson L, Laws ER Jr, Melmed S, Molitch ME, Murad MH, Utz A, Wass JA; Endocrine Society. Acromegaly: an endocrine society clinical practice guideline. J Clin Endocrinol Metab. 2014 Nov;99(11):3933-51. doi: 10.1210/jc.2014-2700. Epub 2014 Oct 30.
PubMed 25356808
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Phase 3 Study of the Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
- ClinicalTrials.gov ID
- NCT03882034
- Acronym / sponsor ID
- 190071
- Conditions
- Pituitary Disease
- Keywords
- Pediatric, Gigantism, Growth Hormone, Hormone, Pituitary Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- 21 Oct 2019 (actual)
- Primary completion
- 8 Sep 2025 (actual)
- Study completion
- 30 Sep 2025 (actual)
- First posted
- 20 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 them
Design:
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 history
Physical exam
Questionnaires
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.
Hand X-ray
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.
...
Detailed description
Study Description:
GH excess is a rare and potentially lethal condition associated with hypersecretion of GH, usually by a pituitary tumor or hyperplasia. When it occurs prior to the complete fusion of growth plates, it leads to pathological tall stature, and it is called gigantism. After the fusion of the growth plates, it is called acromegaly. It may be associated with debilitating cardiovascular disease and/or diabetes. Children and adolescents with gigantism are currently treated with surgery, radiation therapy, and medications, such as octreotide, to reduce hypersecretion of GH; however, these treatments may lack efficacy and have significant side effects. Pegvisomant is a genetically engineered GH-receptor (GHR) antagonist that blocks the action of GH. In adults with acromegaly, pegvisomant has been shown to effectively reduce serum insulin-like growth factor type 1 (IGF-1) concentrations and lead to clinical improvement. However, experience in children and adolescents is limited to a small number of case series.,We propose the initiation of a new protocol at the NICHD, NIH, to treat children and adolescents with GH excess that is refractory to surgical therapy and/or radiation therapy, or in children and adolescents where the above therapies are contraindicated.
Objectives:
PRIMARY OUTCOMES:
- Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
- Determine the safety and tolerability of pegvisomant in children and adolescents with GH excess.
SECONDARY OUTCOMES:
- Percent change of IGF-1 z-score from baseline to end of the study in subgroups of patients receiving monotherapy and patients receiving additional medication that target GH excess.
- Normalization of the IGF-1 for age and sex from baseline to end of study (normal value defined as +/- 2 SD from the mean). Change in growth velocity to a near-normal range (+/- 1 SD) according to the Tanner and Davies growth velocity curves for age, sex, and stage of puberty, when comparing the 6-month period prior to the study drug initiation to the growth velocity between 6 and 12 months on the study drug.
- Improvement in signs and symptoms of GH excess that are common in the pediatric population (headaches, excessive perspiration, fatigue, increased appetite) and the quality of life of the patients from baseline to the end of the study (12 month visit).
- Improvement of the cardiac structure and function: reduction of the left ventricular mass index (LVMi), and change of the left ventricular ejection fraction (EF) on echocardiogram from baseline to the end of the study (12 month visit).
The objectives of the proposed study are to characterize the efficacy of pegvisomant as indicated by adequate control of the IGF-1 levels, the safety profile of the medication in children with GH excess, and to obtain pharmacodynamic data on the effect of pegvisomant on the GH-IGF axis in children. Because pegvisomant does not inhibit GH secretion, serum IGF-1 is the best marker of treatment efficacy. Pharmacokinetic (PK) studies will be performed in a subset of patients. This study will enroll patients with GH excess younger than 18 years and their biological parents.
Endpoints: Safety
Patients who have received at least 1 dose of pegvisomant will be included in the safety evaluations.
Safety will be evaluated by:
- Spontaneously reported adverse events
- Vital signs and periodic physical examinations (performed at the Clinical Center at baseline, 6 months (if possible unless telehealth visit performed) and 12 months after the study enrollment, and at local provider s office at regular intervals between those visits)
- Laboratory tests (performed periodically as indicated by previous studies) including:
- Hematology
- Chemistry
- Lipids
- Liver function panel
- Thyroid function tests
- IGF-1
- Glucose and insulin
- MRI of the pituitary to check for tumor growth at baseline, 6 months, and 1 year
- Annual liver ultrasound
- ECG and echocardiogram at baseline, 6 months (EKG, if possible unless telehealth visit performed) and 1-year to monitor for the presence of cardiac arrhythmias and GH related cardiomyopathy
- Care providers will complete the Gigantism Symptoms Assessment Questionnaire (GSAQ) and Child Health Questionnaire by Landgraf \& Wade for assessment of quality of life.
- Patients with gigantism and diabetes mellitus may require careful monitoring and dose reductions of insulin and/or oral hypoglycemic agents as pegvisomant usually has beneficial effects on glucose metabolism.7 Patients with pre-existing diabetes will continue to monitor their daily blood glucose levels, as previously instructed, and communicate with the investigational team for persistent elevation or decrease of the glucose levels. The changes of the dose of the anti-diabetic drugs and/or insulin will be done with communication of the research team with the local endocrinologist. Patients without pre-existing diabetes will be educated on the signs of hyperglycemia and will get monthly fasting blood glucose level measurements for the first 6 months along with the screening of the liver enzyme levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: PegvisomantIntervention arm, Patient received pegvisomant |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Subjects who are eligible for enrollment must meet the following eligibility criteria:
- Cohort 1: Patients with GH excess
- Males and females 24 months to <18 years at enrollment
- Active GH excess as demonstrated by the following:
- History of inadequate response to trans-sphenoidal surgery or radiation therapy for GH secreting pituitary tumor, or inability to tolerate surgery or radiation therapies or patient deemed inappropriate candidate for surgery and/or pituitary radiation therapy, as determined by review of the medical records by the PI. The evaluation of the patient should be performed at 3 months after the surgery date in order to ensure that there is persistent GH excess after the transsphenoidal resection of the tumor unless there are clear evidence of persistent disease, e.g. residual tumor, based on the PI s assessment. If the patient has received irradiation, there is no minimum time to be considered before enrolling in the study. The effects of radiation therapy take place over many years after receiving it (mean time to remission for stereotactic radiation therapy of 12-60 months), and, thus, a medical therapy is required during that period.
Key exclusion criteria (8 listed)
- Cohort 1: Patients
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Liver function abnormalities (ALT, AST) greater than or equal to 3 x ULN
- Positive pregnancy test in females, current pregnancy and/or female patients who are breastfeeding.
- Patients currently using opioids. Opioids induce altered metabolism of pegvisomant. Since this is a phase 3 study, opioids may affect the PK studies to be performed and, thus, chronic use of opioids (>2 weeks) will be an exclusion criterion.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegvisomantA 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. Adjustment of the dose will occur as per protocol. | Drug | 1 |
Primary outcome
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.
5 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2025.
- Background: PubMed 10770982Trainer PJ, Drake WM, Katznelson L, Freda PU, Herman-Bonert V, van der Lely AJ, Dimaraki EV, Stewart PM, Friend KE, Vance ML, Besser GM, Scarlett JA, Thorner MO, Parkinson C, Klibanski A, Powell JS, Barkan AL, Sheppard MC, Malsonado M, Rose DR, Clemmons DR, Johannsson G, Bengtsson BA, Stavrou S, Kleinberg DL, Cook DM, Phillips LS, Bidlingmaier M, Strasburger CJ, Hackett S, Zib K, Bennett WF, Davis RJ. Treatment of acromegaly with the growth hormone-receptor antagonist pegvisomant. N Engl J Med. 2000 Apr 20;342(16):1171-7. doi: 10.1056/NEJM200004203421604.
- Background: PubMed 26574647Lodish MB, Trivellin G, Stratakis CA. Pituitary gigantism: update on molecular biology and management. Curr Opin Endocrinol Diabetes Obes. 2016 Feb;23(1):72-80. doi: 10.1097/MED.0000000000000212.
- Background: PubMed 25356808Katznelson L, Laws ER Jr, Melmed S, Molitch ME, Murad MH, Utz A, Wass JA; Endocrine Society. Acromegaly: an endocrine society clinical practice guideline. J Clin Endocrinol Metab. 2014 Nov;99(11):3933-51. doi: 10.1210/jc.2014-2700. Epub 2014 Oct 30.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. Protocol is silent on IPD sharing.
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Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 2
A Study to Investigate Mibavademab in Adult Women With Functional Hypothalamic Amenorrhea
Functional Hypothalamic Amenorrhea, FHA
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karim A Calis, Pharm.D. principal investigator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pegvisomant in people with Pituitary Disease, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change of IGF-1 z-score from baseline to end of study (12 month visit). (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03882034, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2025.