Growth Hormone DeficiencyGH-miRNA2NCT06455956
Use of miRNAs in Growth Hormone Deficiency (GHD)
An observational study of Growth Hormone Deficiency, run by University of Parma at 1 site in Italy.
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… Read more about this study
- Who is it for?
- Ages 2 to 15 years
- Conditions the study is for
- Growth Hormone Deficiency
- How many people
- 400 participants
- Where
- 1 siteItaly
- Study dates
- 13 Apr 2023to 1 Mar 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 15 years
- All sexes
- No healthy volunteers
- Child
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.
Who may be able to join
- Children with short stature and suspicion of GHD
- Subjects with organic GH deficiency
- 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
- Isolated idiopathic prepubertal naive GHD subjects on long-acting versus daily GH therapy
Who cannot join
- -Subjects with underlying genetic conditions and chronic diseases
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
University of Parma
Parma, Emilia-Romagna, 43121
Maria E Street, +39 0521 033557, mariaelisabeth.street@unipr.it
About this study
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
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.
Read the 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
- Keywords
- growth hormone, miRNA, growth hormone deficiency, growth hormone treatment, small for gestational age, Turner syndrome, Noonan syndrome, SHOX deficiency
- Medical subject headings
- Dwarfism, Pituitary, Turner Syndrome, Noonan Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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..
Measured: 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.
Measured: 3 years
Secondary outcomes (1)
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.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2024
- Study start13 Apr 2023
- Primary completion31 Mar 2025 (estimated)
- Study completion1 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Parma
- Sponsor type
- University, hospital or other
- Collaborators
- Azienda Ospedaliera Universitaria Policlinico "G. Martino", Ospedali Riuniti Ancona, Federico II University, Azienda Unita Sanitaria Locale di Piacenza, Azienda Ospedaliero Universitaria Policlinico Modena, Azienda USL Reggio Emilia - IRCCS, 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
- Responsible party
- Sponsor
- Investigators
- Maria E Street, MD, PhD, Principal investigator, University of Parma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06455956
- Sponsor's protocol ID
- 821/2022/OSS/UNIPR
- Acronym
- GH-miRNA2
- Status last verified
- May 2024
- First submitted
- 6 Jun 2024
- Last updated
- 13 Jun 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06455956
- Acronym / sponsor ID
- GH-miRNA2 / 821/2022/OSS/UNIPR
- Conditions
- Growth Hormone Deficiency
- Keywords
- growth hormone, miRNA, growth hormone deficiency, growth hormone treatment, small for gestational age, Turner syndrome, Noonan syndrome, SHOX deficiency
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- University of ParmaUniversity, hospital or other
- Collaborators
- Azienda Ospedaliera Universitaria Policlinico "G. Martino", Ospedali Riuniti Ancona, Federico II University, Azienda Unita Sanitaria Locale di Piacenza, Azienda Ospedaliero Universitaria Policlinico Modena, Azienda USL Reggio Emilia - IRCCS, 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
- Study start
- 13 Apr 2023 (actual)
- Primary completion
- 31 Mar 2025 (estimated)
- Study completion
- 1 Mar 2026 (estimated)
- First posted
- 13 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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.
Key inclusion criteria (7 listed)
- Children with short stature and suspicion of GHD
- Subjects with organic GH deficiency
- 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
Key exclusion criteria (1 listed)
- -Subjects with underlying genetic conditions and chronic diseases
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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.
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..
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.
1 secondary outcome
Sex-specific miRNA changes after 3 months on long-acting GH therapy
Time frame: 3 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2024.
- Individual participant dataSharing planned: No
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Enrolling by invitationObservational study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maria E Street, MD, PhD principal investigator
University of Parma
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Growth Hormone Deficiency, run by University of Parma at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 13 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Maria E Street, MD, PhD (+39 0521 033557, mariaelisabeth.street@unipr.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessing new biomarkers for the diagnosis of GHD (time frame: 3 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2024.