Use of miRNAs in Growth Hormone Deficiency (GHD)

Growth Hormone Deficiency Clinical Trial

— GH-miRNA2  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06455956
• Other study ID #: 821/2022/OSS/UNIPR
• Status: Recruiting
• Start date: April 13, 2023
• Est. completion date: March 1, 2026

Study information

• Verified date: May 2024
• Source: University of Parma
• Contact: Maria E Street, MD, PhD
• Phone: +39 0521 033557
• Email: mariaelisabeth.street[@]unipr.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: March 1, 2026
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 15 Years

Eligibility

Inclusion Criteria for aim 1:

• Children with short stature and suspicion of GHD

• Subjects with organic GH deficiency

Exclusion criteria for aim 1:

-Subjects with underlying genetic conditions and chronic diseases

Inclusion Criteria for aim 2-3-5:

• patients having confirmed GHD enrolled for aim 1

• patients with growth failure and born SGA (>4 yr of age)

• patients with Noonan and Turner syndrome and growth failure

• patients with short stature homeobox-containing gene deficiency (SHOXD) and growth failure

Inclusion Criteria for aim 4:

• Isolated idiopathic prepubertal naive GHD subjects on long-acting versus daily GH therapy

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency

Locations

Country	Name	City	State
Italy	University of Parma	Parma	Emilia Romagna

Sponsors (16)

Lead Sponsor

Collaborator

University of Parma

Azienda Ospedaliera Universitaria Policlinico "G. Martino", Azienda Ospedaliero Universitaria Policlinico Modena, Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari, Azienda Unità Sanitaria Locale di Piacenza, Azienda Unità Sanitaria Locale Reggio Emilia, Federico II University, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, IRCCS Azienda Ospedaliero-Universitaria di Bologna, IRCCS Ospedale San Raffaele, Istituto Auxologico Italiano, OSPEDALE CARLO POMA ASST MANTOVA, Ospedali Riuniti Ancona, Ospedali Riuniti di Foggia, Università degli Studi del Piemonte Orientale "Amedeo Avogadro", University of Campania "Luigi Vanvitelli"

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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	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