NCT06455956

Use of miRNAs in Growth Hormone Deficiency (GHD)

New Potential Biomarkers for Diagnosis of Growth Hormone (GH) Deficiency and Early Assessment of Response to GH Treatment for Appropriate Personalization of Therapy

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 400 (estimated)
• Sponsor: University of Parma · Academic / Other
• Sex: All
• Age: 2 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This study aims at improving knowledge about the diagnosis of growth hormone deficiency (GHD) and treatment with growth hormone (GH), with the goal of providing information on the presence of new biomarkers, such as miRNAs, for diagnostic and therapeutic purposes, with the goal of establishing a personalized GH treatment scheme, optimizing resources, reducing costs, and improving outcomes.

Detailed description

In 200 prepubertal and pubertal subjects with the suspicion of GHD, the levels of three specific circulating miRNas will be measured to establish whether they can be used for the diagnosis of GH deficiency. In subjects with isolated idiopathic GHD (IIGHD), short statured patients born SGA, Noonan and Turner syndromes, and SHOX deficiency, we will also assess the changes of the identified miRNAs before and after 3 months on GH therapy to verify an early growth prediction model for growth response at 12 months on treatment. Finally, we plan to identify any differences in these miRNAs at 3 months on treatment using daily GH versus weekly (long-acting) GH.

Conditions

• Growth Hormone Deficiency

Timeline

• Start date: 2023-04-13
• Primary completion: 2025-03-31
• Completion: 2026-03-01
• First posted: 2024-06-13
• Last updated: 2024-06-13

Locations

1 site across 1 country: Italy