The AgRP and GH/IGF-1 Axis in Children

NCT05645211 | Not Yet Recruiting

• 1 other identifier: AAAU4134
• Study Type: observational
• Target: 156
• Locations: 0 countries
• Sites: N/A

Brief 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. 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.

Conditions

Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (2)

• 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

  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

  baseline to 2 months

Secondary Outcomes (5)

• Protocol 1: Correlation of plasma AgRP levels with age

  Baseline (cross-sectional testing at one time point, no intervention)

• Protocol 1: Correlation of plasma AgRP levels with leptin levels

  Baseline (cross-sectional testing at one time point, no intervention)

• Protocol 1: Correlation of plasma AgRP with percent body fat (determined from skinfold thickness)

  Baseline (cross-sectional testing at one time point, no intervention)

• Protocol 2: Change in AgRP levels correlation with change in IGF-1 levels

  Baseline to 2 months

• Protocol 2: Change in AgRP level correlation with pre-treatment leptin levels

  Baseline to 2 months

Study Arms (3)

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.

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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:
• Ambulatory male and female children aged 5-9 years who are prepubertal
• Normal weight at birth
• Growth failure
• Peak GH response to 2 GH stimulation tests \< 10 ng/ml
• Normal renal and liver function
• 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.
• 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:
• Ambulatory male and female children aged 5-9 years who are prepubertal

Sponsors & Collaborators

• Columbia University: lead

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Central Study Contacts

Pamela Freda

CONTACT

2123052254; puf1@cumc.columbia.edu

Rosa Lazarte

CONTACT

2123054921; rl2345@cumc.columbia.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Medicine at CUIMC

Study Record Dates

• First Submitted: November 30, 2022
• First Posted: December 9, 2022
• Study Start (Estimated): June 10, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: August 26, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share