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RECRUITING
NCT06668805
PHASE2

A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

Official title: A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment.

Key Details

Gender

All

Age Range

3 Years - 11 Years

Study Type

INTERVENTIONAL

Enrollment

54

Start Date

2024-11-22

Completion Date

2041-09

Last Updated

2026-02-27

Healthy Volunteers

No

Interventions

DRUG

Vosoritide Injection

Modified recombinant human C-type natriuretic peptide Vosoritide

Locations (24)

Children's Hospital of Orange County Main Campus - Orange

Orange, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Nicklaus Children's Hospital

Miami, Florida, United States

St. Luke's Children's Endocrinology and Diabetes

Boise, Idaho, United States

Kentucky Children's Hospital

Lexington, Kentucky, United States

New York Medical College

Boston, Massachusetts, United States

M Health Fairview Pediatric Specialty Clinic - Explorer

Minneapolis, Minnesota, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Children's Hospital at Montefiore

The Bronx, New York, United States

Atrium Health Carolinas Medical Center

Charlotte, North Carolina, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Children's Medical Center Dallas

Dallas, Texas, United States

University of Texas Health Science Center at Houston (UT Health)

Houston, Texas, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Hôpital de la Timone

Marseille, Bouches-du-Rhône, France

Angers University Hospital Center

Angers, Maine-et-Loire, France

CHU de Toulouse - Hôpital des Enfants

Toulouse, Occitanie, France

South Paris University Hospitals - Bicetre Hospital

Le Kremlin-Bicêtre, Paris, France

Hôpital Robert-Debré

Paris, Paris, France

Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

Florence, Florence, Italy

IRCCS Istituto Giannina Gaslini

Genova, Genoa, Italy

Central University Hospital of Asturias

Oviedo, Principality of Asturias, Spain