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Achondroplasia clinical trials

View clinical trials related to Achondroplasia.

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NCT ID: NCT06168201 Recruiting - Achondroplasia Clinical Trials

A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States

VISTA
Start date: February 21, 2023
Phase:
Study type: Observational [Patient Registry]

This is an observational study of a pediatric cohort with achondroplasia in the United States. This cohort consists of both individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats: Participant-mediated access to electronic health records which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data The primary study population will include individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa)

NCT ID: NCT06079398 Recruiting - Achondroplasia Clinical Trials

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Start date: January 23, 2024
Phase: Phase 2
Study type: Interventional

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.

NCT ID: NCT06067425 Recruiting - Clinical trials for Osteochondrodysplasia

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

upreACH-2
Start date: October 10, 2023
Phase: Phase 2
Study type: Interventional

This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.

NCT ID: NCT05603936 Recruiting - Clinical trials for Growth Hormone Deficiency

Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4

Start date: March 1, 2022
Phase:
Study type: Observational

The aim of the study is the adaption, implementation and validation of the instrument for the investigation of the short stature specific quality of life (QoLISSY) for children (age 0-4 years) with achondroplasia (ACH), Small for Gestational Age (SGA) and Growth Hormone Deficiency (GHD) from a parental perspective.

NCT ID: NCT05353192 Recruiting - Achondroplasia Clinical Trials

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

Start date: September 23, 2022
Phase: Phase 4
Study type: Interventional

To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

NCT ID: NCT05328050 Recruiting - Achondroplasia Clinical Trials

Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)

OMPR-Ach/Hy
Start date: September 1, 2021
Phase:
Study type: Observational [Patient Registry]

This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.

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. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

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: 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 <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.