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Achondroplasia clinical trials

View clinical trials related to Achondroplasia.

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NCT ID: NCT05659719 Completed - Achondroplasia Clinical Trials

A Study to Learn About Recifercept in Patients With Achondroplasia

Start date: October 19, 2022
Phase:
Study type: Observational

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. The study will include data already collected from a recifercept clinical trial and data collected from a separate study of achondroplasia. This study will compare patient experiences and will help the investigators determine if the study medicine, recifercept, is effective.

NCT ID: NCT03875534 Completed - Achondroplasia Clinical Trials

A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

Start date: June 19, 2019
Phase:
Study type: Observational

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. This is a natural history study and no study medication will be administered.

NCT ID: NCT03872531 Completed - Achondroplasia Clinical Trials

Lifetime Impact Study for Achondroplasia

LISA
Start date: March 31, 2019
Phase:
Study type: Observational

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 look at the impact on the following in individuals with achondroplasia versus a normative population: - Quality of life - Clinical burden - Healthcare resource use - Socio-economic burden - Psychosocial burden Up to 175 subjects will be enrolled in sites in Argentina, Colombia and Brazil.

NCT ID: NCT03780153 Completed - Achondroplasia Clinical Trials

The Norwegian Adult Achondroplasia Study

Start date: March 1, 2017
Phase:
Study type: Observational

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 function, and work participation.

NCT ID: NCT03583697 Completed - Achondroplasia Clinical Trials

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Start date: May 23, 2018
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT03449368 Completed - Achondroplasia Clinical Trials

Lifetime Impact of Achondroplasia Study in Europe-LIAISE

LIAISE
Start date: December 17, 2017
Phase:
Study type: Observational

Observational study looking at the burden of illness in achondroplasia subjects aged 5-70. The study will include a 5 year review of historical clinical data as well as a single point collection of questionnaire data to look at the impact on the following in individuals with achondroplasia versus a normative population: - Quality of life - Clinical burden - Healthcare resource use - Socio-economic burden - Psychosocial burden Up to 300 subjects will be included in sites in Germany, Spain, Italy, Sweden, Austria and Denmark

NCT ID: NCT03197766 Completed - Achondroplasia Clinical Trials

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Start date: December 12, 2016
Phase: Phase 3
Study type: Interventional

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.

NCT ID: NCT02055157 Completed - Achondroplasia Clinical Trials

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

ACH
Start date: January 13, 2014
Phase: Phase 2
Study type: Interventional

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 achondroplasia.

NCT ID: NCT01603095 Completed - Achondroplasia Clinical Trials

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Start date: April 2012
Phase:
Study type: Observational

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. No study drug is administered.

NCT ID: NCT01590446 Completed - Achondroplasia Clinical Trials

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

Start date: February 2012
Phase: Phase 1
Study type: Interventional

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. Information about any side effects that may occur will also be collected.