Clinical trials: Hypochondroplasia
Genetic Disorder, Achondroplasia
A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
This study is conducted in Japan. The aim of the study is to evaluate the efficacy of somatropin (Norditropin®) on adult height (cm) in patients with achondroplasia / hypochondroplasia enrolled in the GH-1941 study (NCT01516229).
- Trial name
- NCT01435629
- Testing
- somatropin
- Where
- Japan
- Volunteers
- 81 people
- Sponsor
- Novo Nordisk A/S
Short Stature
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.
- Trial name
- NCT04219007
- Testing
- Vosoritide
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Andrew Dauber
Hypochondroplasia
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
This study is conducted to describe the efficacy and safety of recombinant human growth hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.
- Trial name
- NCT01111019
- Testing
- Recombinant human growth hormone (r-hGH)
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Achondroplasia, Hypochondroplasia
Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)
This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.
- Trial name
- OMPR-Ach/Hy (NCT05328050)
- Testing
- Registry
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Hypochondroplasia
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to < 36 months over a 52-week period.
- Trial name
- NCT07126262
- Testing
- Vosoritide
- Where
- 26 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia, Hypochondroplasia
C-Type Natriuretic Peptide and Achondroplasia
Achondroplasia and hypochondroplasia are the most common forms of dwarfism. Recent studies have shown that a small hormone called C-type natriuretic peptide (CNP) is an important regulator of linear growth.
- Trial name
- NCT01541306
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Nemours Children's Clinic
Hypochondroplasia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
- Trial name
- CANOPY-HCH-3 (NCT06455059)
- Testing
- Vosoritide
- Where
- 23 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
- Trial name
- CANOPY-HCH-EXT (NCT07073014)
- Testing
- Vosoritide
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 140 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia, Hypochondroplasia
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
- Trial name
- SKY-MOT3 (NCT07388966)
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 140 people
- Sponsor
- SYSNAV
Hypochondroplasia
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.
- Trial name
- NCT06212947
- Where
- 43 sites in United States, Argentina and 9 more
- Volunteers
- 400 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company