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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06455956

Acronym

GH-miRNA2

Enrollment

400

Registered

2024-06-13

Start date

2023-04-13

Completion date

2026-03-01

Last updated

2024-06-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Growth Hormone Deficiency

Keywords

growth hormone, miRNA, growth hormone deficiency, growth hormone treatment, small for gestational age, Turner syndrome, Noonan syndrome, SHOX deficiency

Brief 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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to 15 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Assessing new biomarkers for the diagnosis of GHD	3 years	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.
Measuring miRNA changes after 3 months on GH therapy	3 years	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.
Multiple linear regression models	3 years	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..
Measuring miRNA changes between daily and long-acting GH therapy	3 years	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.

Secondary

Measure	Time frame	Description
Sex-specific miRNA changes after 3 months on long-acting GH therapy	3 years	Any differences between males and females in miRNAs expression after 3 months of long-acting GH therapy will be evaluated.

Countries

Italy

Contacts

Primary ContactMaria E Street, MD, PhD

mariaelisabeth.street@unipr.it+39 0521 033557

Outcome results