Growth Hormone DeficiencyNCT05645211
The AgRP and GH/IGF-1 Axis in Children
An observational study of Growth Hormone Deficiency, run by Columbia University.
Trial summary
Recent data support the existence of a GH-Agouti-related peptide (AgRP) axis. The neuropeptide AgRP promotes food intake and has important effects on energy homeostasis. Recent evidence suggest that GH stimulates AgRP and AgRP may mediate some of GH's important nutritional and metabolic effects. Read more about this study
- Who is it for?
- Ages 5 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Growth Hormone Deficiency
- How many people
- 156 participants
- Where
- No sites listed yet
- Study dates
- 10 Jun 2027to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 5 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: Healthy children: 140 healthy children (70 females and 70 males) between the ages of 5-17 yr. will be studied; 7-8 children per year of this age range will be studied. They will be representative of the diverse racial and ethnic mix of children who receive their medical care at our medical center.Children will be recruited from among the healthy children visiting primary care Pediatricians at our medical center. GH deficient and idiopathic short stature children: 16 children (8 males, 8 females) between the ages of 5 - 9 yr. who are pre-pubertal and who plan to start GH treatment for isolated GH deficiency or idiopathic short stature. They will be representative of the diverse racial and ethnic mix of our hospital's patient population.
Who may be able to join
- Ambulatory male and female children aged 5-17 years
- Normal weight at birth
- Height between the 3rd and 99th percentiles of the mean as per the CDC growth percentiles.
- Ambulatory male and female children aged 5-9 years who are prepubertal
- Normal weight at birth
- Growth failure
Show all 14Show fewer
- Peak GH response to 2 GH stimulation tests < 10 ng/ml
- Normal renal and liver function
- Ambulatory male and female children aged 5-9 years who are prepubertal
- Normal weight at birth
- Height >2.25 SD below mean for age
- Peak GH response to 2 stimulation tests >10 ng/ml or normal IGF-1 and IGFBP-3 levels
- No prior supplemental growth hormone exposure
- Normal renal and liver function
Who cannot join
- Genetic defects, chronic illnesses.
- Current prescription medication use
- Use of glucocorticoids, thyroid hormone or medications that may affect the GH-IGF-1 axis within 6 months of study entry.
- Children with GH deficiency:
- Multiple pituitary hormone deficiencies,
- GH deficiency or poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome.
Show all 12Show fewer
- History of diabetes or malignancy
- Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry.
- Children with idiopathic Short Stature:
- Poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome.
- History of diabetes or malignancy
- Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Agouti-related Peptide (AgRP) and the GH/IGF-1 Axis in Children
Recent data support the existence of a GH-Agouti-related peptide (AgRP) axis. The neuropeptide AgRP promotes food intake and has important effects on energy homeostasis. Recent evidence suggest that GH stimulates AgRP and AgRP may mediate some of GH's important nutritional and metabolic effects. main goals of this project are to characterize, for the first time, plasma levels of AgRP in children and to determine how these relate to GH and IGF-1 levels, age, body composition, clinical and other endocrine parameters. To accomplish this, we will conduct two studies, one being a cross-sectional study that will measure AgRP levels in 140 healthy children ages 5-17 and the second being a prospective study that will measure the change in plasma AgRP levels in response to GH treatment in 16 children who receive this as part of their clinical care for GH deficiency or short stature.
Read the detailed description
Protocol 1 This will be a cross-sectional study in 140 healthy children. Participation will include one visit that will take place between 8-9 am and after a fast from midnight the night before.
Procedures at the visit will include:
- Review of medical history
- Anthropometrics measurements: Weight, height, waist and hip circumferences.
- Collection of information from medical record including growth records and physical examination findings including features relevant to pubertal stage in all children and onset of menses in females.
- Assessment of pubertal status and Tanner stage based on physical examination.
- Collection of information on diet, activity level and sleep.
- Blood Sampling: venous blood will be sampled from a peripheral vein for measurement of AgRP, GH, IGF-1, leptin, SOb-R, triglycerides, insulin, glucose, testosterone(males), estradiol(females), DHEAS and cortisol levels. Insulin and glucose levels will be used to assess insulin resistance by HOMA \& QUICKI.
Protocol 2 This will be a prospective study in 16 children who will be studied before and at 4 time points (1 week, 2 weeks, 1 month and 2 months) after starting GH treatment as part of their clinical care. This protocol will study subjects Groups 2 and 3. Growth hormone will not be prescribed as part of this study. Children will be treated clinically with GH as prescribed by their Pediatric Endocrinologist for FDA approved indications and according to standard guidelines for dosing for treatment of GH deficiency in children.
Each visit that will take place between 8-9 am and after a fast from midnight the night before.
Procedures at each visit will include:
- Review of medical history
- Anthropometrics measurements: Weight, height, waist and hip circumferences, skinfold thicknesses.
- Collection of information from medical record including growth records and physical examination findings including features relevant to pubertal stage in all children and onset of menses in females. GH dose and compliance will be recorded at follow up visits.
- Collection of information on diet, activity level and sleep.
- Blood Sampling: Venous blood will be sampled at a peripheral vein for:
Baseline (pre-GH treatment): measurement of AgRP, GH, IGF-1, IGFBP-3, leptin, SOb-R, triglycerides, insulin and glucose, testosterone(males), estradiol(females), DHEAS and cortisol levels.
Follow up visits on growth hormone: measurement of AgRP, IGF-1, IGFBP-3, leptin, SOb-R, triglycerides, insulin, glucose and cortisol levels.
Insulin and glucose levels will be used to assess insulin resistance by HOMA \& QUICKI.
- Conditions
- Growth Hormone Deficiency
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Healthy children | 140 healthy children (70 females and 70 males) between the ages of 5-17 yr. will be studied; 7-8 children per year of this age range will be studied. They will be representative of the diverse racial and ethnic mix of children who receive their medical care at our medical center. Inclusion Criteria: 1. Ambulatory male and female children aged 5-17 years 2. Normal weight at birth 3. Height between the 3rd and 99th percentiles of the mean as per the CDC growth percentiles. Exclusion Criteria: 1. Genetic defects, chronic illnesses. 2. Current prescription medication use 3. Use of glucocorticoids, thyroid hormone or medications that may affect the GH-IGF-1 axis within 6 months of study entry. | |
| Group 2: Children with GH deficiency | 16 children (8 males, 8 females) between the ages of 5 - 9 yr. who are pre-pubertal and who plan to start GH treatment for isolated GH deficiency or idiopathic short stature. They will be representative of the diverse racial and ethnic mix of our hospital's patient population. GH deficiency: Inclusion Criteria: 1. Ambulatory male and female children aged 5-9 years who are prepubertal 2. Normal weight at birth 3. Growth failure 4. Peak GH response to 2 GH stimulation tests < 10 ng/ml 5. Normal renal and liver function Exclusion criteria: 1. Multiple pituitary hormone deficiencies, 2. GH deficiency or poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome. 3. History of diabetes or malignancy 4. Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry. | |
| Group 3: Children with short stature | Idiopathic Short Stature: Inclusion Criteria: 1. Ambulatory male and female children aged 5-9 years who are prepubertal 2. Normal weight at birth 3. Height >2.25 SD below mean for age 4. Peak GH response to 2 stimulation tests >10 ng/ml or normal IGF-1 and IGFBP-3 levels 5. No prior supplemental growth hormone exposure 6. Normal renal and liver function Exclusion criteria: 1. Poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome. 2. History of diabetes or malignancy 3. Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Protocol 1: Correlation of Plasma AgRP levels with IGF-1 levels
Plasma AgRP levels correlation with IGF-1 levels Protocol 1: Correlation of Plasma AgRP levels with IGF-1 levels
Measured: Baseline (cross-sectional at single time point, no intervention)
Protocol 2: Change in plasma AgRP levels with GH therapy
Change in plasma AgRP levels from pre-GH therapy to post-GH therapy
Measured: baseline to 2 months
Secondary outcomes (5)
Protocol 1: Correlation of plasma AgRP levels with age
Correlation of plasma AgRP levels with age
Measured: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 1: Correlation of plasma AgRP levels with leptin levels
Correlation of plasma AgRP levels with leptin levels
Measured: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 1: Correlation of plasma AgRP with percent body fat (determined from skinfold thickness)
Correlation of plasma AgRP with percent body fat (determined from skinfold thickness)
Measured: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 2: Change in AgRP levels correlation with change in IGF-1 levels
Correlation of change in AgRP levels with change in IGF-1 levels from baseline to 2 months of GH therapy
Measured: Baseline to 2 months
Protocol 2: Change in AgRP level correlation with pre-treatment leptin levels
Correlation of change in AgRP level from baseline to 2 months of GH therapy with pre-treatment leptin levels
Measured: Baseline to 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2022
- Study start10 Jun 2027 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 156 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pamela U. Freda, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05645211
- Sponsor's protocol ID
- AAAU4134
- Status last verified
- Sep 2026
- First submitted
- 30 Nov 2022
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Agouti-related Peptide (AgRP) and the GH/IGF-1 Axis in Children
- ClinicalTrials.gov ID
- NCT05645211
- Acronym / sponsor ID
- AAAU4134
- Conditions
- Growth Hormone Deficiency
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 156 participants (estimated)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Study start
- 10 Jun 2027 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 9 Dec 2022 (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.
Recent data support the existence of a GH-Agouti-related peptide (AgRP) axis. The neuropeptide AgRP promotes food intake and has important effects on energy homeostasis. Recent evidence suggest that GH stimulates AgRP and AgRP may mediate some of GH's important nutritional and metabolic effects. main goals of this project are to characterize, for the first time, plasma levels of AgRP in children and to determine how these relate to GH and IGF-1 levels, age, body composition, clinical and other endocrine parameters. To accomplish this, we will conduct two studies, one being a cross-sectional study that will measure AgRP levels in 140 healthy children ages 5-17 and the second being a prospective study that will measure the change in plasma AgRP levels in response to GH treatment in 16 children who receive this as part of their clinical care for GH deficiency or short stature.
Detailed description
Protocol 1 This will be a cross-sectional study in 140 healthy children. Participation will include one visit that will take place between 8-9 am and after a fast from midnight the night before.
Procedures at the visit will include:
- Review of medical history
- Anthropometrics measurements: Weight, height, waist and hip circumferences.
- Collection of information from medical record including growth records and physical examination findings including features relevant to pubertal stage in all children and onset of menses in females.
- Assessment of pubertal status and Tanner stage based on physical examination.
- Collection of information on diet, activity level and sleep.
- Blood Sampling: venous blood will be sampled from a peripheral vein for measurement of AgRP, GH, IGF-1, leptin, SOb-R, triglycerides, insulin, glucose, testosterone(males), estradiol(females), DHEAS and cortisol levels. Insulin and glucose levels will be used to assess insulin resistance by HOMA \& QUICKI.
Protocol 2 This will be a prospective study in 16 children who will be studied before and at 4 time points (1 week, 2 weeks, 1 month and 2 months) after starting GH treatment as part of their clinical care. This protocol will study subjects Groups 2 and 3. Growth hormone will not be prescribed as part of this study. Children will be treated clinically with GH as prescribed by their Pediatric Endocrinologist for FDA approved indications and according to standard guidelines for dosing for treatment of GH deficiency in children.
Each visit that will take place between 8-9 am and after a fast from midnight the night before.
Procedures at each visit will include:
- Review of medical history
- Anthropometrics measurements: Weight, height, waist and hip circumferences, skinfold thicknesses.
- Collection of information from medical record including growth records and physical examination findings including features relevant to pubertal stage in all children and onset of menses in females. GH dose and compliance will be recorded at follow up visits.
- Collection of information on diet, activity level and sleep.
- Blood Sampling: Venous blood will be sampled at a peripheral vein for:
Baseline (pre-GH treatment): measurement of AgRP, GH, IGF-1, IGFBP-3, leptin, SOb-R, triglycerides, insulin and glucose, testosterone(males), estradiol(females), DHEAS and cortisol levels.
Follow up visits on growth hormone: measurement of AgRP, IGF-1, IGFBP-3, leptin, SOb-R, triglycerides, insulin, glucose and cortisol levels.
Insulin and glucose levels will be used to assess insulin resistance by HOMA \& QUICKI.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1: Healthy children | – | –140 healthy children (70 females and 70 males) between the ages of 5-17 yr. will be studied; 7-8 children per year of this age range will be studied. They will be representative of the diverse racial and ethnic mix of children who receive their medical care at our medical center. Inclusion Criteria: 1. Ambulatory male and female children aged 5-17 years 2. Normal weight at birth 3. Height between the 3rd and 99th percentiles of the mean as per the CDC growth percentiles. Exclusion Criteria: 1. Genetic defects, chronic illnesses. 2. Current prescription medication use 3. Use of glucocorticoids, thyroid hormone or medications that may affect the GH-IGF-1 axis within 6 months of study entry. |
| Group 2: Children with GH deficiency | – | –16 children (8 males, 8 females) between the ages of 5 - 9 yr. who are pre-pubertal and who plan to start GH treatment for isolated GH deficiency or idiopathic short stature. They will be representative of the diverse racial and ethnic mix of our hospital's patient population. GH deficiency: Inclusion Criteria: 1. Ambulatory male and female children aged 5-9 years who are prepubertal 2. Normal weight at birth 3. Growth failure 4. Peak GH response to 2 GH stimulation tests < 10 ng/ml 5. Normal renal and liver function Exclusion criteria: 1. Multiple pituitary hormone deficiencies, 2. GH deficiency or poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome. 3. History of diabetes or malignancy 4. Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry. |
| Group 3: Children with short stature | – | –Idiopathic Short Stature: Inclusion Criteria: 1. Ambulatory male and female children aged 5-9 years who are prepubertal 2. Normal weight at birth 3. Height >2.25 SD below mean for age 4. Peak GH response to 2 stimulation tests >10 ng/ml or normal IGF-1 and IGFBP-3 levels 5. No prior supplemental growth hormone exposure 6. Normal renal and liver function Exclusion criteria: 1. Poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome. 2. History of diabetes or malignancy 3. Use of glucocorticoids or medications known to affect the GH-IGF-1 axis within 6 months of study entry. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Healthy children: 140 healthy children (70 females and 70 males) between the ages of 5-17 yr. will be studied; 7-8 children per year of this age range will be studied. They will be representative of the diverse racial and ethnic mix of children who receive their medical care at our medical center.Children will be recruited from among the healthy children visiting primary care Pediatricians at our medical center. GH deficient and idiopathic short stature children: 16 children (8 males, 8 females) between the ages of 5 - 9 yr. who are pre-pubertal and who plan to start GH treatment for isolated GH deficiency or idiopathic short stature. They will be representative of the diverse racial and ethnic mix of our hospital's patient population.
Key inclusion criteria (14 listed)
- Ambulatory male and female children aged 5-17 years
- Normal weight at birth
- Height between the 3rd and 99th percentiles of the mean as per the CDC growth percentiles.
- Ambulatory male and female children aged 5-9 years who are prepubertal
- Normal weight at birth
Key exclusion criteria (12 listed)
- Genetic defects, chronic illnesses.
- Current prescription medication use
- Use of glucocorticoids, thyroid hormone or medications that may affect the GH-IGF-1 axis within 6 months of study entry.
- Multiple pituitary hormone deficiencies,
- GH deficiency or poor growth associated with any acute or chronic medical condition such as renal disease or Turner's syndrome.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Protocol 1: Correlation of Plasma AgRP levels with IGF-1 levels
Time frame: Baseline (cross-sectional at single time point, no intervention)
Plasma AgRP levels correlation with IGF-1 levels Protocol 1: Correlation of Plasma AgRP levels with IGF-1 levels
Protocol 2: Change in plasma AgRP levels with GH therapy
Time frame: baseline to 2 months
Change in plasma AgRP levels from pre-GH therapy to post-GH therapy
5 secondary outcomes
Protocol 1: Correlation of plasma AgRP levels with age
Time frame: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 1: Correlation of plasma AgRP levels with leptin levels
Time frame: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 1: Correlation of plasma AgRP with percent body fat (determined from skinfold thickness)
Time frame: Baseline (cross-sectional testing at one time point, no intervention)
Protocol 2: Change in AgRP levels correlation with change in IGF-1 levels
Time frame: Baseline to 2 months
Protocol 2: Change in AgRP level correlation with pre-treatment leptin levels
Time frame: Baseline to 2 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Growth Hormone Deficiency
RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
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.
Questions about this trial
What is this clinical trial studying?
An observational study of Growth Hormone Deficiency, run by Columbia University.
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 14 inclusion and 12 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: opening soon, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pamela Freda (2123052254, puf1@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Protocol 1: Correlation of Plasma AgRP levels with IGF-1 levels (time frame: Baseline (cross-sectional at single time point, no intervention)). 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?
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 25 Sep 2026.