Clinical trials: Achondroplasia
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
- Trial name
- NCT04085523
- Testing
- TransCon CNP
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 57 people
- Sponsor
- Ascendis Pharma A/S
Achondroplasia
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height.
- Trial name
- NCT03989947
- Testing
- Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 73 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
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…
- Trial name
- NCT04638153
- Testing
- Recifercept
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 60 people
- Sponsor
- Pfizer
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
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…
- Trial name
- NCT05116046
- Testing
- Recifercept
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Pfizer
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH.
- Trial name
- NCT03875534
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 260 people
- Sponsor
- Ascendis Pharma A/S
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia
Purpose of the study: The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China.
- Trial name
- NCT05246033
- Testing
- TransCon CNP
- Where
- 5 sites in China
- Volunteers
- 24 people
- Sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.
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
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.
- Trial name
- NCT06842355
- Testing
- TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg, TYRA-300 0.375 mg/kg
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 92 people
- Sponsor
- Tyra Biosciences, Inc
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Achondroplasia
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
- Trial name
- NCT05813314
- Testing
- BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
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
- Trial name
- NCT04554940
- Testing
- vosoritide
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
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.
- Trial name
- NCT03794609
- Where
- 57 sites in United States, Australia and 12 more
- Volunteers
- 315 people
- Sponsor
- Pfizer
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 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
Achondroplasia
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose.
- Trial name
- NCT01590446
- Testing
- BMN 111, Normal Saline
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial.
- Trial name
- AttaCH (NCT05929807)
- Testing
- TransCon CNP
- Where
- 19 sites in United States, Australia and 10 more
- Volunteers
- 140 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
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
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