Clinical trials: Achondroplasia
Achondroplasia
The Norwegian Adult Achondroplasia Study
The study aims to investigate the Norwegian adult achondroplasia population regarding degree and extent of symptoms and clinical manifestations and how this population manages in daily life, including demographics, physical…
- Trial name
- NCT03780153
- Testing
- No intervention
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Sunnaas Rehabilitation Hospital
Achondroplasia
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin.
- Trial name
- NCT01603095
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 363 people
- Sponsor
- BioMarin Pharmaceutical
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
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide…
- Trial name
- NCT06079398
- Testing
- Navepegritide
- Where
- 18 sites in United States, Australia and 13 more
- Volunteers
- 72 people
- Sponsor
- Ascendis Pharma A/S
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
- Trial name
- NCT05353192
- Testing
- Recombinant human growth hormone
- Where
- China
- Volunteers
- 39 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Achondroplasia
Lifetime Impact Study for Achondroplasia
Observational study looking at the burden of illness in achondroplasia subjects aged 3 and above. The study will include a 3 year review of historical clinical data as well as a single point collection of questionnaire data to…
- Trial name
- LISA (NCT03872531)
- Where
- 4 sites in Argentina, Brazil and 1 more
- Volunteers
- 173 people
- Sponsor
- BioMarin Pharmaceutical
Osteochondrodysplasia
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
- Trial name
- upreACH-2 (NCT06067425)
- Testing
- SAR442501
- Where
- 9 sites in Australia, China and 3 more
- Volunteers
- 16 people
- Sponsor
- Sanofi