Use of miRNAs in Growth Hormone Deficiency (GHD) (GH-miRNA2)

June 6, 2024 updated by: University of Parma

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
400

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Italy
    • Emilia Romagna
      • Parma, Emilia Romagna, Italy, 43121

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All patients will be investigated in routine practice during the normal workup for short stature and stunted growth. Treatment will be also delivered accordind to standard care indications and as established by regulatory agencies in Italy.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Assessing new biomarkers for the diagnosis of GHD
Time Frame: 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.
3 years
Measuring miRNA changes after 3 months on GH therapy
Time Frame: 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.
3 years
Multiple linear regression models
Time Frame: 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..
3 years
Measuring miRNA changes between daily and long-acting GH therapy
Time Frame: 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.
3 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Sex-specific miRNA changes after 3 months on long-acting GH therapy
Time Frame: 3 years
Any differences between males and females in miRNAs expression after 3 months of long-acting GH therapy will be evaluated.
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of Parma

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Federico II University
University of Campania "Luigi Vanvitelli"
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Istituto Auxologico Italiano
Azienda Unita Sanitaria Locale Reggio Emilia
Azienda Ospedaliera Universitaria Policlinico "G. Martino"
Università degli Studi del Piemonte Orientale "Amedeo Avogadro"
Azienda Unita Sanitaria Locale di Piacenza
Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari
Ospedali Riuniti Ancona
Ospedali Riuniti di Foggia
OSPEDALE CARLO POMA ASST MANTOVA
IRCCS Ospedale San Raffaele
Azienda Ospedaliero Universitaria Policlinico Modena

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Maria E Street, MD, PhD, University of Parma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 13, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 6, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 13, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 13, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 821/2022/OSS/UNIPR

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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