Clinical trials: Achondroplasia
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period…
- Trial name
- ApproaCH (NCT05598320)
- Testing
- Navepegritide (TransCon CNP)
- Where
- 10 sites in United States, Australia and 5 more
- Volunteers
- 84 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
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
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 Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 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
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
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
Achondroplasia
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with…
- Trial name
- ACH (NCT02055157)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical