Prospective Clinical Assessment Study in Children With Achondroplasia

Clinical Trial Summary

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.

See also
Status	Clinical Trial	Phase
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Recruiting	`NCT05328050` - Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)
Completed	`NCT05659719` - A Study to Learn About Recifercept in Patients With Achondroplasia
Active, not recruiting	`NCT04554940` - A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia	Phase 2
Completed	`NCT01435629` - A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin	N/A
Enrolling by invitation	`NCT06164951` - A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia	Phase 3
Completed	`NCT01516229` - Special Survey for Long Term Application	N/A
Completed	`NCT03872531` - Lifetime Impact Study for Achondroplasia
Active, not recruiting	`NCT05598320` - A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia	Phase 2/Phase 3
Terminated	`NCT05813314` - Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants	Phase 1
Recruiting	`NCT04265651` - Study of Infigratinib in Children With Achondroplasia	Phase 2
Recruiting	`NCT05603936` - Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
Completed	`NCT03780153` - The Norwegian Adult Achondroplasia Study
Active, not recruiting	`NCT04085523` - A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia	Phase 2
Enrolling by invitation	`NCT05929807` - A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia	Phase 2/Phase 3
Not yet recruiting	`NCT06433557` - A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia	Phase 2
Completed	`NCT03875534` - A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Terminated	`NCT03794609` - Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Completed	`NCT03583697` - A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia	Phase 2
Active, not recruiting	`NCT03989947` - An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia	Phase 2

NCT number	NCT04035811
Study type	Observational
Source	QED Therapeutics, Inc.
Contact	QED Therapeutics, Inc.
Phone	1-877-280-5655
Email	PROPELstudyinfo@QEDTX.com
Status	Recruiting
Phase
Start date	August 12, 2019
Completion date	June 2026

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms