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severe-short-stature Clinical Trials

A listing of severe-short-stature medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 28 clinical trials
L Lauren Samz

A Study of TYRA-300 in Children With Achondroplasia: BEACH301

The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.

3 - 10 years of age All Phase 2
G Gabor Szinnai, Prof. MD, PhD

Prospective Evaluation of OptiThyDose

The aim of this multicentric, randomised, two-arms and single-blinded clinical trial is to prospectively evaluate OptiThyDose for Congenital hypothyroidism (CH) and Graves' disease (GD).

- 18 years of age All Phase N/A
J Joshua Smith, Dr

A Study to Assess Growth in Children With Idiopathic Short Stature

Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.

2 - 16 years of age All Phase N/A
M Maria Saleco Montejo, MD

A Natural History Study in Children With a Type II Collagen Disorder With Short Stature

There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well as data to support identification of possible risk factors. This study aims to …

- 12 years of age All Phase N/A
H Ho Seong Kim, M.D., Ph.D

Long-term Safety and Effectiveness of Growtropin®-II Treatment in Children With Short Stature

This study evaluates long-term safety and effectiveness of Growtropin®-II treatment in children with short stature.

2 years of age All Phase N/A
c chunxiu Gong, doctor

The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg

In order to further observe the long-term safety and effectiveness of real-world polyethylene glycol-recombinant human growth hormone(PEG-rhGH) treatment of GHD, idiopathic short stature, and SGA in children, explore and analyze the factors affecting the efficacy of PEG-rhGH and the height prediction model after treatment, etc., collect and analyze more scientifically …

14 - 18 years of age All Phase N/A

IDMet (RaDiCo Cohort) (RaDiCo-IDMet)

The goal of this observational study is to describe the natural history of imprinting disorders (IDs) according to their metabolic profile in all patients (adults and children) affected with an ID regardless of the severity of the disease, with a molecular characterization, with a signed informed consent for all subjects, …

years of age All Phase N/A

Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children

The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal Failure), SGA (Small for Gestational Age), and ISS (Idiopathic Short Stature).

2 years of age All Phase N/A
O Omar Sherbini, MPH

The Myelin Disorders Biorepository Project

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade …

years of age All Phase N/A
C CoRDS Team

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures …

years of age All Phase N/A

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