Biological Age in Children With GH Deficiency Undergoing Hormone Replacement Therapy

NCT06294860 | Completed

• 1 other identifier: 01C317
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of the study is to evaluate the epigenetic age in children with GH deficiency, before and after 6 months of treatment with growth hormone replacement therapy. The secondary objective is to correlate the epigenetic age with the auxometric and biochemical parameters used in the clinical-endocrinological practice. The results of the study will be useful to set up the clinical and biochemical follow-up of the hormone replacement therapy with rhGH and to understand the biomolecular mechanisms at the base of the debated "anti" or "pro" aging action of GH, the most important anabolic hormone of the human organism.

Conditions

Growth Hormone Deficiency; Short Stature

Keywords

Growth hormone deficiency; Replacement therapy; Recombinant growth hormone; Short stature; Biological age; Epigenetic

Outcome Measures

Primary Outcomes (1)

• Measurement of biological (epigenetic) age

  The biological age (epigenetic) is measured in basal condition (T0) and after 6 months of treatment with recombinant growth hormone with two algorithms (Zbiec-Piekarska and Daunay), based on DNA methylation level in specific loci. To have an estimate of the epigenetic age (biological) that is independent of the chronological age, we use age acceleration, for whose calculation we apply a linear regression model with the chronological age as independent variable and the epigenetic age as dependent variable; the difference between the observed value and the predicted value from the model is the age acceleration due to a properly epigenetic effect. In case the epigenetic age is higher than the chronological age, the age acceleration has a positive value expressed in years, otherwise negative.

  Baseline and after 6 months of substitutive treatment with recombinant growth hormone

Study Arms (1)

Hormone replacement therapy with rhGH

Each subject will be treated with hormone replacement therapy with rhGH with a dose of 0.025-0.035 mg/kg body weight per day (or 0.7-1.0 mg/ m2 body surface area per day) for a period of 6 months.

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Children with isolated growth hormone deficiency

You may qualify if:

• children of both sexes
• age 5-15 years affected by isolated GH deficiency according to the criteria provided by note 39 AIFA for this pathology (short stature: ≤ -3 DS or ≤ -2 DS; growth rate/year ≤ -1.0 DS per age and sex evaluated at a distance of at least six months; GH peak at two different pharmacological stimulation tests \< 8 ng/ml).

You may not qualify if:

• \- presence of organic hypothalamic-pituitary pathologies (diagnosed through encephalic NMR).

Sponsors & Collaborators

• Istituto Auxologico Italiano: lead

Study Sites (1)

Istituto Auxologico Italiano

Milan, 20145, Italy

Location

Biospecimen

Retention: SAMPLES WITH DNA

From the leucocytes isolated from the samples taken at T0 and T6, the DNA is extracted. The DNA methylation is performed with sodium bisulfite and PCR- Pyrosequencing.

MeSH Terms

Conditions

Dwarfism, Pituitary; Dwarfism

Condition Hierarchy (Ancestors)

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; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Alessandro Sartorio, MD

  Istituto Auxologico Italiano

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 28, 2024
• First Posted: March 6, 2024
• Study Start: June 19, 2023
• Primary Completion: March 30, 2025
• Study Completion: March 30, 2025
• Last Updated: September 3, 2025

Record last verified: 2025-09

Locations

IT (1)