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Achondroplasia clinical trials

View clinical trials related to Achondroplasia.

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NCT ID: NCT04638153 Recruiting - 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.

NCT ID: NCT04554940 Recruiting - Achondroplasia Clinical Trials

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Start date: October 10, 2020
Phase: Phase 2
Study type: Interventional

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

NCT ID: NCT04265651 Recruiting - Achondroplasia Clinical Trials

Study of Infigratinib in Children With Achondroplasia

Start date: March 10, 2020
Phase: Phase 2
Study type: Interventional

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion.

NCT ID: NCT04184817 Recruiting - Achondroplasia Clinical Trials

Radiological Analysis on Patients With Achondroplasia Disorder

Achondroplasia
Start date: February 15, 2020
Phase:
Study type: Observational

Achondroplasia is a genetic disorder characterized by disproportionate short stature. It affects about 1 in 2500 live births in the world. The cause of Achondroplasia was identified to be a gain-of-function mutations in the fibroblast growth receptor 3 (FGFR3). In these children compression of the spinal cord at the foramen magnum stenosis can occur in early childhood which, can lead to central sleep apnea. It can lead to morbidity and mortality. A surgical intervention may be indicated in patients who present a foramen magnum stenosis. However, surgical indications are still under discussion. The objective of this retrospective study is to analyse the degree of stenosis and its clinical tolerance/evolution from radiological data monitored at the Hospital Femme Mère Enfant.

NCT ID: NCT04085523 Enrolling by invitation - Achondroplasia Clinical Trials

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

Start date: June 24, 2020
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT04035811 Recruiting - Achondroplasia Clinical Trials

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Start date: August 12, 2019
Phase:
Study type: Observational

This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.

NCT ID: NCT03989947 Enrolling by invitation - Achondroplasia Clinical Trials

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

Start date: June 12, 2019
Phase: Phase 2
Study type: Interventional

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. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

NCT ID: NCT03875534 Recruiting - 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: NCT03866200 Recruiting - 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.