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 5 clinical trials
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 …

  • 30 Jul, 2021
  • 11 locations
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

  • 01 Aug, 2021
  • 3 locations
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
  • 30 Jul, 2021
  • 5 locations
Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum Cockayne Syndrome or Trichothiodystrophy

Four rare genetic diseases, xeroderma pigmentosum (XP), Cockayne syndrome (CS), the XP/CS complex and trichothiodystrophy (TTD) have defective DNA excision repair although only XP has increased cancer susceptibility. We plan to perform careful clinical examination of selected patients with XP, XP/CS, CS, or TTD and follow their clinical course. We …

cancer prevention
  • 24 Jul, 2021
  • 1 location
Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype Correlation

, short stature, and bowing of the long bones. There is no known cure for OI so researchers want to learn more about it. Objectives To obtain a natural history of the

dental exam
connective tissue disorder
bone mineral density
short stature
  • 23 Jul, 2021
  • 1 location