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Achondroplasia clinical trials

View clinical trials related to Achondroplasia.

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NCT ID: NCT05813314 Terminated - Achondroplasia Clinical Trials

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Start date: March 15, 2023
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT05116046 Terminated - Achondroplasia Clinical Trials

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

Start date: December 24, 2021
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT04638153 Terminated - Achondroplasia Clinical Trials

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

Start date: December 2, 2020
Phase: Phase 2
Study type: Interventional

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, 3 6, 9 & 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements & patient/caregiver quality of life questionnaires Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study. A PK cohort will include 12 participants who will randomly receive a single dose of 3 mg/kg of Phase 2 study (process 1c) formulation and a single dose of 3 mg/kg of the proposed Phase 3 (process 2) study formulation in a cross over study. Dose of the cohort could be changed due to emerging safety and efficacy data in the study.

NCT ID: NCT03866200 Terminated - Clinical trials for Pseudoachondroplasia

Resveratrol Trial for Relief of Pain in Pseudoachondroplasia

Start date: June 29, 2019
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine if self-administered oral resveratrol can dampen joint pain for individuals with pseudoachondroplasia compared to placebo. Another goal of this study is to evaluate side effects in this population.

NCT ID: NCT03794609 Terminated - Achondroplasia Clinical Trials

Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Start date: June 15, 2018
Phase:
Study type: Observational

This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments Children's information will be collected in the registry for a maximum of 5 years.