Vesiculogenesis in Children With GH Deficiency (VESCIGHTP)

NCT05759624 | Active Not Recruiting

• 1 other identifier: 01C211
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of the study is to evaluate the size and derivational profile of the extracellular vescicles (EV) generated in children with GH deficiency, undergoing hormone replacement therapy with rhGH. Secondary objectives is to correlate vesiculogenesis with auxometric and biochemical parameters used in clinical-endocrine practice in the evaluation of short stature. The results of the study will provide useful information to more rationally set up the clinical and biochemical follow-up of hormone replacement therapy with rhGH, as well as to understand the molecular and cellular mechanisms underlying the multi-systemic action of GH, the most important anabolic hormone of the human organism.

Conditions

GH Deficiency

Keywords

Growth; GH deficiency; Children; Short stature; Extracellular vescicles

Outcome Measures

Primary Outcomes (2)

• Size of extracellular vesicles

  Change in size of extracellular vesicles after rhGH treatment

  Baseline and after 6 months

• Derivational profile of extracellular vesicles

  Change in derivational profile of extracellular vesicles after rhGH treatment

  Baseline and after 6 months

Secondary Outcomes (4)

• Auxometric parameters - height

  Baseline and after 6 months

• Auxometric parameters - weight

  Baseline and after 6 months

• Biochemical parameters - glycemia

  Baseline and after 6 months

• Biochemical parameters - Insulin-like growth factor I (IGF-I)

  Baseline and after 6 months

Study Arms (1)

Hormone replacement therapy with rhGH

Drug: rhGH

Interventions

rhGH DRUG

Hormone replacement therapy with rhGH at a dose of 0.025-0.035 mg/kg of body weight per day (or 0.7-1.0 mg/m2 of body surface area per day).

Hormone replacement therapy with rhGH

Eligibility Criteria

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

Study Population

10 children of both sexes affected by isolated GH deficiency will be recruited according to the criteria set out in AIFA note 39 for this pathology (short stature: ≤ - 3 SD or ≤ -2 SD and growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart and peak GH at two different stimulus pharmacological tests \< 8 ng/ml).

You may qualify if:

• short stature: ≤ - 3 SD or ≤ -2 SD
• growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart - peak GH at two different stimulus pharmacological tests \< 8 ng/ml

You may not qualify if:

• \- presence of organic pathologies at the hypothalamic-pituitary level (assessed by performing brain MRI).

Sponsors & Collaborators

• Istituto Auxologico Italiano: lead

Study Sites (1)

Istituto Auxologico Italiano IRCCS

Milan, 20145, Italy

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum/plasma samples

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 Design

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

Study Record Dates

• First Submitted: February 26, 2023
• First Posted: March 8, 2023
• Study Start: June 24, 2022
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: October 2, 2024

Record last verified: 2024-10

Locations

IT (1)