Use of miRNAs in Growth Hormone Deficiency (GHD)
NCT Number: NCT06455956
Acronym: GH-miRNA2
Status: RECRUITING
Study Type: OBSERVATIONAL
Conditions: Growth Hormone Deficiency
Sponsor: University of Parma
Collaborators: Azienda Ospedaliera Universitaria Policlinico "G. Martino"|Ospedali Riuniti Ancona|Federico II University|Azienda Unità Sanitaria Locale di Piacenza|Azienda Ospedaliero Universitaria Policlinico Modena|Azienda Unità Sanitaria Locale Reggio Emilia|Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari|Ospedali Riuniti di Foggia|IRCCS Ospedale San Raffaele|Università degli Studi del Piemonte Orientale "Amedeo Avogadro"|IRCCS Azienda Ospedaliero-Universitaria di Bologna|Istituto Auxologico Italiano|OSPEDALE CARLO POMA ASST MANTOVA|University of Campania "Luigi Vanvitelli"|Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Sex: ALL
Age: CHILD
Enrollment: 400
Study Design: Observational Model: |Time Perspective: p
Start Date: 2023-04-13
Completion Date: 2026-03-01
Locations: University of Parma, Parma, Emilia Romagna, 43121, Italy

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.

Primary Outcome Measures: Assessing new biomarkers for the diagnosis of GHD, The 1st objective will consist in the enrolment of 200 subjects, nationwide, undergoing standard routine work-up for the diagnosis of GHD. The specific miRNAs will be measured at the time of both stimulation tests in order to include subjects with both confirmed and unconfirmed GHD and guarantee reproducibility; in addition, subjects with organic GH deficiency will be included., 3 years|Measuring miRNA changes after 3 months on GH therapy, The 2nd objective will consist in measuring these specific miRNAs before and at 3 months on treatment in 200 subjects with isolated idiopathic GHD(IIGHD), and in 60 short statured patients born SGA, in 60 having Noonan and Turner syndromes, and in 30 with SHOX deficiency, according to current indications to verify whether changes are confirmed in pubertal versus prepubertal subjects and in conditions other than IIGHD., 3 years|Multiple linear regression models, The 3rd objective will consist in investigating the major determinants of height variations between 0 and 6 months, 0 and 12 months and the variance of growth rate variation between 0 and 6 months including the levels and/or changes in the specific miRNAs at baseline and at 3 months on treatment by using multiple linear regression models to establish early prediction models of growth response.., 3 years|Measuring miRNA changes between daily and long-acting GH therapy, The 4th objective will consist in measuring these specific miRNAs before and at 3 months on treatment in children with IIGHD being treated with daily versus longacting GH. Findings will be compared with those in the IIGHD group receiving daily GH treatment as an exploratory task, thus, only a small number of subjects will be enrolled, and anyway as many as possible., 3 years
Secondary Outcome Measures: Sex-specific miRNA changes after 3 months on long-acting GH therapy, Any differences between males and females in miRNAs expression after 3 months of long-acting GH therapy will be evaluated., 3 years