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Short Stature clinical trials

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NCT ID: NCT06295341 Recruiting - Short Stature Clinical Trials

Short Stature and Psychological Well-being

PSICOSHORT
Start date: May 10, 2023
Phase:
Study type: Observational

The first aim of the present study is to evaluate the psychological impact of the condition of short stature (family) in a sample of Italian children, comparing them with subjects of normal stature, measuring their levels of psychological well-being, psychological distress, quality of health-related life and any behavioral issues. The secondary objective is to study the psychological impact evaluated with the tests described below (see methods section) in children with GH deficiency and the effects of replacement therapy (6 months) with GH from recombinant DNA.

NCT ID: NCT06294860 Recruiting - Clinical trials for Growth Hormone Deficiency

Biological Age in Children With GH Deficiency Undergoing Hormone Replacement Therapy

ETABIOGHD
Start date: June 19, 2023
Phase:
Study type: Observational

The primary objective of the study is to evaluate the epigenetic age in children with GH deficiency, before and after 6 months of treatment with growth hormone replacement therapy. The secondary objective is to correlate the epigenetic age with the auxometric and biochemical parameters used in the clinical-endocrinological practice. The results of the study will be useful to set up the clinical and biochemical follow-up of the hormone replacement therapy with rhGH and to understand the biomolecular mechanisms at the base of the debated "anti" or "pro" aging action of GH, the most important anabolic hormone of the human organism.

NCT ID: NCT05849389 Recruiting - Turner Syndrome Clinical Trials

Vosoritide for Short Stature in Turner Syndrome

Start date: March 2024
Phase: Phase 2
Study type: Interventional

Turner syndrome (TS) is characterized by a missing whole or part of the second sex chromosome in a phenotypic female, resulting in short stature due to haploinsufficiency of the short-stature homeobox-containing (SHOX) gene. Growth hormone (GH) is an approved therapy for this condition, although not associated with GH deficiency, and benefits are modest. Vosoritide, a C-type natriuretic peptide (CNP) analog, targets chondrocytes within the growth plate leading to increased cell proliferation and hypertrophy. We hypothesize that patients with TS and short stature will respond to vosoritide treatment leading to increased growth velocity. This study will enroll pre-pubertal girls with TS who are either naïve to GH or have had a poor response to GH therapy. All subjects will be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes. Annualized growth velocity (AGV) on vosoritide will be compared to AGV in the 6-18 months prior to initiation of vosoritide based on historical data available in the medical record. Subjects with a positive response to therapy will be given the option to continue in the extension phase of the study during which they will continue to receive vosoritide until growth cessation.

NCT ID: NCT05829252 Recruiting - Growth Disorders Clinical Trials

Testing the Feasibility of a Novel Growth Monitoring Smartphone App

Start date: March 18, 2021
Phase:
Study type: Observational

The goal of this clinical trial is to test the acceptability, feasibility, accuracy and precision of the 'GrowthMonitor' smartphone app in a pilot population.

NCT ID: NCT04774653 Recruiting - Short Stature Clinical Trials

Hypo-mineralization of Primary and Permanent Teeth in a Group of Children With Stunted Growth.A Cross Sectional Study.

Start date: April 2021
Phase:
Study type: Observational

The second primary molar (SPM) development start at the same time as development of the first permanent molars (FPM) and permanent incisors so any systemic disturbance - causing stunted growth -occur , will result in hypo-mineralization of SPM as well as FPM and permanent incisors (Butler 1967, Weerheijm and Mejàre 2003). The literature shows no previous studies that discuss the association between hypo- mineralized second primary molar (HSPM), MIH and the stunted growth in children. aim: Estimate Prevalence of HSPM and MIH in stunted children.Evaluate the association between HSPM, MIH and the stunted growth in a group of Egyptian children. • The diagnostic criteria for MIH established based on the European Academy of Pediatric Dentistry criteria (Weerheijm and Mejàre 2003) while diagnostic criteria for HSPM was established by (Elfrink et al. 2008).

NCT ID: NCT04754516 Completed - Short Stature Clinical Trials

Diagnosis of Short Stature in Children : Study of a Cohort of Small-sized Consulting Patients

Start date: January 1, 2021
Phase:
Study type: Observational

Many patients consult in pediatric endocrinology because of their small size. In the majority of cases, this growth delays can be explained by a hormonal, gastroenterological cause, or a chronic disease. Sometimes the reason for stunted growth can be constitutional bone disease, a genetic cause of short stature that is still underdiagnosed. The investigators wish to describe and take stock of the various additional analyzes carried out and the various diagnoses made in patients who consulted in endocrino-pediatrics at the Montpellier University Hospital due to their short stature, in 2017 and 2018, in order to better screen patients with constitutional bone disease

NCT ID: NCT04476433 Completed - Asthma Clinical Trials

Intervention in Chronic Pediatric Patients and Their Families.

FACTORADAPT
Start date: February 4, 2019
Phase: N/A
Study type: Interventional

This project consists of a psychological intervention in patients and their families with different chronic diseases in order to carry out a comparative study between medical pathologies to know which are the protective or risk variables for the adaptation to the disease.

NCT ID: NCT04275141 Completed - Diabetes Mellitus Clinical Trials

Mauriac Syndrome: Isotopic Techniques and Genetic Analysis

Start date: January 17, 2022
Phase: N/A
Study type: Interventional

Mauriac syndrome (MS) is an entity of individuals combining poorly controlled diabetes mellitus type 1, short stature and glycogenic hepatopathy. Thus, the functional significance of Mauriac syndrome for glucose metabolism remains disputed, and whether genetic defects in glycogen metabolism contribute to glycogenic hepatopathy in MS remains to be clarified.Coupling the genetic analysis of targeted genes involved in glucose regulation with a dynamic exploration will eventually determine if a genetic abnormality leads to the disease and explains the nature of the phenotype.

NCT ID: NCT04244123 Completed - Short Stature Clinical Trials

Web-based Adherence Information Integrated Nurse-led Monitoring Clinic

WAIN-MC
Start date: January 24, 2020
Phase:
Study type: Observational

Non-adherence is a recognized problem with growth hormone treatment in children. In this study, we aim to utilize web-based information derived from easypod growth hormone injection devices and easypod connect devices in a nurse-led telephone clinic to improve adherence and therefore optimize growth. Our primary aim is to test height SDS change over a 12 month period. Our secondary aims are to test adherence, acceptance/satisfaction and qualitative assessment.

NCT ID: NCT04219007 Active, not recruiting - Short Stature Clinical Trials

Vosoritide for Selected Genetic Causes of Short Stature

Start date: August 4, 2020
Phase: Phase 2
Study type: Interventional

Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.