Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Summary

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 63 participants will be offered to continue at the previously received dose of Recifercept either Low Dose Medium Dose High Dose or at the therapeutic dose once it is identified. Participants will attend the clinic monthly for 24 months. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires.

Timeline

Start

2021-12-24

Primary completion

2022-12-16

Completion

2023-03-30

Outcome

Outcome not reported

Stopped: “The study was terminated due to lack of efficacy at any of the tested doses on 18th November 2022. The decision to terminate the study is not related to a safety concern.”

Drugs

Indications