Vosoritide for Selected Genetic Causes of Short Stature

Status Active, not recruiting

Clinical Trial Summary

Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04219007
Study type	Interventional
Source	Children's National Research Institute
Contact
Status	Active, not recruiting
Phase	Phase 2
Start date	August 4, 2020
Completion date	June 1, 2035

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02826902` - Effect of Anesthesia on Quality of Recovery in Patients Undergoing Correctional Tibial Osteotomy - A Randomized Controlled Trial	N/A
Completed	`NCT00235599` - The IGFBP-3 Stimulation Test: A New Tool for the Diagnosis of Growth Hormone Deficiency in Children.	N/A
Completed	`NCT01911260` - Weekly Zinc Chelate Supplementation on Children's Growth	Phase 2/Phase 3
Completed	`NCT02137538` - Aromatase Inhibitor Growth Study: Letrozole vs. Anastrozole	Phase 4
Recruiting	`NCT01934270` - Growth Hormone Secretion Following the Anaerobic Exercise	N/A
Completed	`NCT00443144` - D3-GHR Polymorphism and Turner Syndrome	N/A
Recruiting	`NCT06295341` - Short Stature and Psychological Well-being
Recruiting	`NCT05849389` - Vosoritide for Short Stature in Turner Syndrome	Phase 2
Recruiting	`NCT05829252` - Testing the Feasibility of a Novel Growth Monitoring Smartphone App
Withdrawn	`NCT03323177` - Long Term Effects of Nutritional Supplementation on Final Height	N/A
Completed	`NCT02389803` - Evaluating the Effect of Nutritional Supplementation on Growth of Short and Lean Adolescents Boys	N/A
Recruiting	`NCT06294860` - Biological Age in Children With GH Deficiency Undergoing Hormone Replacement Therapy
Recruiting	`NCT03123003` - Bone Age Assessment in Children Using Ultrasound Compared to Wrist X-ray	N/A
Completed	`NCT03575221` - Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype Correlation
Terminated	`NCT01237041` - Free Fatty Acids, Body Weight, and Growth Hormones Secretion in Children	Phase 1/Phase 2
Completed	`NCT04244123` - Web-based Adherence Information Integrated Nurse-led Monitoring Clinic
Recruiting	`NCT01901666` - Assessment Of Gh-Igf-1 Axis In Children With Chronic Myelogenous Leukemia (CML) In Remission	Phase 4
Withdrawn	`NCT01970800` - The Role of Igf-1 Generation Test in Diagnosis and Treatment of Short Stature	N/A
Completed	`NCT00830141` - Study of the Molecular Basis in the Pathophysiology of Food Intake and Growth in Children	N/A
Completed	`NCT00562705` - Effects of Growth Hormone (GH) Treatment on Eating Regulation	N/A