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 18 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
  • 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
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
Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)

This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH. Subjects will be randomized in a 2:1 ratio to Treatment (Tirosint®-SOL) or Control (conventional therapy with levothyroxine sodium crushed tablets).

  • 21 Apr, 2022
  • 1 location
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism

This clinical trial will use growth hormone as a novel treatment for Phelan-McDermid syndrome (PMS) and idiopathic autism. A double-blind, placebo-controlled crossover trial design will be used in 30 children with idiopathic autism and 15 children with PMS to evaluate the the effects of growth hormone on visual evoked potentials …

  • 10 Aug, 2022
PREPP: Preventing Postpartum Depression (PREPP)

The primary aim of this study is to determine if a behavioral intervention targeting maternal caregiving of young infants can increase infant sleep and reduce fuss/cry behavior, and thereby (1) reduce the incidence and/or severity of postpartum maternal depression and (2) improve the quality of the mother-infant interaction and subsequent …

behavior therapy
Accepts healthy volunteers
depressed mood
  • 20 Apr, 2022
  • 1 location
Eltrombopag for People With Fanconi Anemia

Background Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new …

neutrophil count
platelet count
fanconi syndrome
  • 24 Jul, 2022
  • 1 location
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.

  • 19 Apr, 2022
  • 19 locations
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments Children's information will be collected in the registry for a maximum of 5 years.

  • 29 Apr, 2022
  • 36 locations