Gene Expression in Monocytes of Growth Hormone Deficient Children

NCT04352712 | Unknown

• 1 other identifier: GHUSalerno
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

The Growth hormone (GH) is mainly synthesized in the anterior portion of the pituitary gland and has an effect on different body areas. Secreted in the circulatory stream, growth hormone reaches the liver and here stimulates the secretion of somatomedin C better known as insulin-like growth factor 1 (IGF), which constitutes its main anabolic effector. Growth hormone deficiency (GHD) is characterized by a delay in the statural growth in children and is correlated with a worsening of body composition, cognitive functions, lipid metabolism, bone mineralization, cardiac performance and exercise in adults. Recombinant GH (rhGH) replacement therapy can correct these alterations and therefore improve the quality of life in treated patients, and accelerate growth in children. The optimal dosage of rhGH varies for each patient, as the response to treatment suffers from considerable inter-individual variability. To date, IGF1 is the only available biomarker whose plasma levels correlate with replacement therapy. It is important to underline how somatomedin C does not provide information about the optimal posology of rhGH for each patient in order, therefore, to predict its adverse events and efficacy. In addition, it has been shown that the effects mediated by the somatotropic hormone on some tissues are direct, therefore independent of the action of IGF1, whose plasma levels are not, in this case, predictive of therapeutic response. For this reason, it is therefore necessary to identify a more specific biomarker capable of monitoring the efficacy, individual responsiveness and any adverse events in patients receiving somatotropic hormone. The GH receptor (GHR) is expressed in several cells, including monocytes. It is therefore possible that the response of monocytes to the somatotropic hormone partially mirrors that of the chondrocyte and other cell types. Given the difficulty of obtaining osteomuscular biopsies or specific body areas in which GH mediates its biological action, the published works have identified the specific cell line in which to study the molecular effects of the hormone in monocytes, thanks to their easy accessibility and high number of GHR. In consideration of this, the investigators propose to stimulate monocytes of healthy and GHD children in vitro with rhGH and through next generation sequencing to identify the characteristic gene expression profile. The GH responsive genes identified with this study can be used for correlation studies on the response to rhGH treatment.

Conditions

Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (1)

• gene expression in monocytes of GHD children

  analysis of RNA-seq of monocytes of healthy and GHD children

  one year

Secondary Outcomes (1)

• gene expression in monocytes modulated by GH

  one year

Study Arms (2)

Healthy Children

healthy children, untreated, donors of monocytes

Other: blood sampling

GHD children

GHD children, untreated, donors of monocytes

Other: blood sampling

Interventions

blood sampling OTHER

blood sampling

GHD children; Healthy Children

Eligibility Criteria

• Age: 6 Years - 10 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

primary care clinic

You may qualify if:

• Height lower the 3th percentile

You may not qualify if:

• Any endocrinopathy
• Liver, kidney or haemolymphopoietic system disorders
• Celiac disease or other chronic malabsorption conditions
• Genetic syndromes (such as Turner's S., Cystic fibrosis or Down S.)
• Drug therapies interfering with growth

Sponsors & Collaborators

• University of Salerno: lead

Study Sites (1)

Department of Medicine and Surgery, Univeristy of Salerno

Baronissi, Salerno, 84081, Italy

RECRUITING

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Blood Specimen Collection

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

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Mario Vitale, MD

  University of Salerno

  STUDY DIRECTOR

Central Study Contacts

Mario Vitale, MD

CONTACT

393482652847; mavitale@unisa.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 15, 2020
• First Posted: April 20, 2020
• Study Start: April 15, 2019
• Primary Completion: April 15, 2021
• Study Completion: April 15, 2021
• Last Updated: May 14, 2020

Record last verified: 2020-04

Locations

IT (1)