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.

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Found 38 clinical trials
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

supplementation versus GH & placebo on growth parameters in 64 children with Idiopathic Short Stature (ISS) after the second year of GH treatment. Participants will be randomly assigned either to the

  • 11 Mar, 2022
  • 1 location
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) …

tyrosine kinase inhibitor
  • 21 Apr, 2022
  • 18 locations
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

Approximately 63 participants will be randomized to one of three doses to receive Recifercept either Low Dose Medium Dose High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 & then Month 2, 3 6, 9 & 12. Assessments include safety, blood …

  • 10 Aug, 2022
  • 10 locations
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this open-label extension (OLE) study for up to an additional 24 months of treatment. Approximately …

  • 11 Aug, 2022
  • 5 locations
Albright Hereditary Osteodystrophy: Natural History, Growth, and Cognitive/Behavioral Assessments

FD003409, IND 67148 or those who meet the criteria of idiopathic short stature or SGA. We are also evaluating neurocognitive and psychosocial functioning in participants with AHO in order to

  • 23 Jan, 2022
  • 1 location
A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature

strategy is used to treat children with ISS (Idiopathic Short Stature). The long-term efficacy and safety of the investigational product are evaluated, which can provide more scientific and reliable

growth hormone
  • 25 Feb, 2022
  • 6 locations
Vosoritide for Selected Genetic Causes of Short Stature

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

saddan syndrome
  • 10 May, 2021
  • 1 location
Metabolic Study of Cockayne Syndrome (METABO-CS)

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 …

Accepts healthy volunteers
  • 24 Feb, 2022
  • 1 location
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia

Purpose of the study: The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China. Study Treatments: TransCon CNP is an investigational (new) drug, which means that it is currently being …

  • 23 Mar, 2022
  • 2 locations
Extension Study of Infigratinib in Children With Achondroplasia (ACH)

This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Treatment-naïve subjects …

  • 26 Apr, 2022
  • 4 locations