Back to Trials
NCT06668805PHASE2Recruiting

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

BioMarin Pharmaceutical

Start Date

11/22/2024

Completion Date

9/1/2041

Summary

The purpose of this study in children with 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.

Detailed Description

This is a Phase 2, randomized, multicenter, study of vosoritide in children with Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, and an analysis of the impact of vosoritide on final adult height (FAH).

Eligibility Criteria

Age Range: 3 years to 11 years

Inclusion Criteria: 1. Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form 2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome. 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Previous or current hGH treatment for short stature associated with their condition. 6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts Exclusion Criteria: 1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia. 2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 3. Bone age advanced beyond chronological age by more than 2 years. 4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 5. Have an unstable condition likely to require surgical intervention during the study. 6. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays. 7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Interventions

DRUG

Vosoritide Injection

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Noonan Syndrome

Locations

Childrens Hospital Los Angeles

Los Angeles, California 90027

United States

Children's Hospital of Orange County Main Campus - Orange

Orange, California 92868

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

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

Wilmington, Delaware 19803

United States

Children's National Medical Center

Washington D.C., District of Columbia 20010

United States

Nicklaus Children's Hospital

Miami, Florida 33155

United States

Emory University School of Medicine

Atlanta, Georgia 30322

United States

St. Luke's Children's Endocrinology and Diabetes

Boise, Idaho 83712

United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois 60611

United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana 46202

United States

University of Iowa

Iowa City, Iowa 52242

United States

Kentucky Children's Hospital

Lexington, Kentucky 40506

United States

New York Medical College

Boston, Massachusetts 02115

United States

M Health Fairview Pediatric Specialty Clinic - Explorer

Minneapolis, Minnesota 55454

United States

Icahn School of Medicine at Mount Sinai

New York, New York 10029

United States

Children's Hospital at Montefiore

The Bronx, New York 10467

United States

Atrium Health Carolinas Medical Center

Charlotte, North Carolina 28203

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 45229-3026

United States

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232-2578

United States

Children's Medical Center Dallas

Dallas, Texas 75235

United States

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

Houston, Texas 77030

United States

Royal North Shore Hospital

Saint Leonards, New South Wales 2065

Australia

Childrens Health Queensland Hospital and Health Service

South Brisbane, Queensland 4101

Australia

Murdoch Children's Research Institute

Parkville, Victoria 3052

Australia

M.A.G.I.C. Clinic

Calgary, Alberta T2E 7H7

Canada

Hôpital de la Timone

Marseille, Bouches-du-Rhône 13385

France

Angers University Hospital Center

Angers, Maine-et-Loire 49 933

France

CHU de Toulouse - Hôpital des Enfants

Toulouse, Occitanie

France

South Paris University Hospitals - Bicetre Hospital

Le Kremlin-Bicêtre, Paris 94270

France

Hôpital Robert-Debré

Paris, Paris 75019

France

Universitätsklinikum des Saarlandes

Homburg, Saarland 66421

Germany

Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

Florence, Florence Firenze

Italy

IRCCS Istituto Giannina Gaslini

Genova, Genoa 16147

Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, 00 168

Italy

Central University Hospital of Asturias

Oviedo, Principality of Asturias 33011

Spain