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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 26 clinical trials
R Rachael Powers

Study of Infigratinib in Children With Achondroplasia

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) …

3 - 11 years of age Both Phase 2
F Feihong Luo

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

2 - 10 years of age Both Phase 4

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < …

- 2 years of age Both Phase 2
K Kimberly Boucher, RN

Vosoritide for Short Stature in Turner Syndrome

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 …

3 - 11 years of age Female Phase 2
M Myriam Durand

Metabolic Study of Cockayne Syndrome

Cockayne syndrome (CS) is related to defective DNA transcription and/or repair and belongs to the family of Nucleotide Excision Repair. It is an autosomal recessive multisystemic disorder characterized by mental retardation, microcephaly, severe growth failure with lipoatrophia, sensorial impairment, cutaneous photosensitivity, dental decay, enophtalmios. The disease is progressive causing severe …

6 - 100 years of age Both Phase N/A
M Moshe Phillip, Prof

The Effect of Nutritional Formula Supplementation on Linear Growth of Growth Hormone (GH) Treated Prepubertal Children With Idiopathic Short Stature (ISS) After 2 Years From the Beginning of GH-therapy

The proposed study is a double blind, randomized, placebo controlled study. The aim of the study is to evaluate the effect of combined growth hormone (GH) treatment &nutritional formula supplementation versus GH & placebo on growth parameters in 64 children with Idiopathic Short Stature (ISS) after the second year of …

5 - 10 years of age Both Phase N/A

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.

3 - 16 years of age Both Phase N/A

A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

The study doctor will collect information from participants with Idiopathic Short Stature, who were treated with growth hormone for at least a year when they were children, before they reached puberty. The word "Idiopathic" refers to "unknown cause", and as such the study participants have/had short stature with no identifiable …

3 - 11 years of age Both Phase N/A
K Karen Heath

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 Both Phase N/A

Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed

The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at …

- 18 years of age Both Phase N/A

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