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Found 38 clinical trials
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
  • 26 Dec, 2021
  • 17 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 …

  • 22 Oct, 2021
  • 11 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
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

stimulation test
hormones levels
saddan syndrome
cardiofaciocutaneous syndrome
  • 10 May, 2021
  • 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
  • 21 Jan, 2021
  • 2 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

  • 05 Aug, 2021
  • 3 locations
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 …

  • 20 Sep, 2021
  • 1 location
Nutritional Stimulation of Growth in Children With Short Stature

Short stature is a frequent reason for referral to a pediatric endocrinology clinic. Short stature is especially prevalent among those with failure to thrive (whose weight is significantly below

  • 29 Sep, 2021
  • 1 location
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 …

  • 12 Dec, 2021
  • 1 location
Mauriac Syndrome: Isotopic Techniques and Genetic Analysis

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

glycogenic hepatopathy
injection of insulin
  • 21 Jul, 2021
  • 2 locations