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

NCT06668805 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 111-211
• Study Type: interventional
• Target: 54
• Locations: 5 countries
• Sites: 24

Brief 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.

Conditions

Short Stature Homeobox- Containing Gene SHOX Deficiency; Noonan Syndrome; Turner Syndrome

Keywords

Short Stature; Musculoskeletal Diseases; Bone Diseases; Developmental Endocrine System Diseases Natriuretic Peptide, C-Type

Outcome Measures

Primary Outcomes (1)

• Change from baseline in Annualized Growth Velocity (AGV)

  At 6 months

Secondary Outcomes (34)

• Incidence of treatment-emergent adverse events

  Until the end of the study, up to 15 years

• Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome

  Every 12 months through the end of the study, up to 15 years

• Incidence of cardiac conditions requiring discontinuation of study treatment

  Every 12 months through the end of the study, up to 15 years

• Change from baseline in height

  Every 6 months through the end of the study, up to 15 years

• Change from baseline in height Z-score

  Every 6 months through the end of the study, up to 15 years

Study Arms (3)

Vosoritide Dose 1 - Low Dose

EXPERIMENTAL

Drug: Vosoritide - Dose 1 Injection • Experimental Drug Lyophilized powder for reconstitution

Drug: Vosoritide Injection

Vosoritide Dose 2 - Medium Dose

EXPERIMENTAL

Drug: Vosoritide - Dose 2 Injection • Experimental Drug Lyophilized powder for reconstitution

Drug: Vosoritide Injection

Vosoritide Dose 3- High Dose

EXPERIMENTAL

Drug: Vosoritide Dose 3 Injection • Experimental Drug Lyophilized powder for reconstitution

Drug: Vosoritide Injection

Interventions

Vosoritide Injection DRUG

Modified recombinant human C-type natriuretic peptide Vosoritide

Vosoritide Dose 1 - Low Dose; Vosoritide Dose 2 - Medium Dose; Vosoritide Dose 3- High Dose

Eligibility Criteria

• Age: 3 Years - 11 Years
• Gender Eligibility Details: Participants must be ≥ 3 years old, and \<11 years old (female) or \<12 years old (males) at the time of signing the informed consent form (ICF).
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
• 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.
• Tanner Stage 1, at time of signing the ICF.
• Previous or current hGH treatment for short stature associated with their condition.
• 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

You may not qualify if:

• Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
• 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.
• Bone age advanced beyond chronological age by more than 2 years.
• Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
• Have an unstable condition likely to require surgical intervention during the study.
• 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.
• Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
• Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (24)

Children's Hospital of Orange County Main Campus - Orange

Orange, California, 92868, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

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

Wilmington, Delaware, 19803, United States

RECRUITING

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

NOT YET RECRUITING

St. Luke's Children's Endocrinology and Diabetes

Boise, Idaho, 83712, United States

RECRUITING

Kentucky Children's Hospital

Lexington, Kentucky, 40506, United States

RECRUITING

New York Medical College

Boston, Massachusetts, 02115, United States

RECRUITING

M Health Fairview Pediatric Specialty Clinic - Explorer

Minneapolis, Minnesota, 55454, United States

RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

RECRUITING

Atrium Health Carolinas Medical Center

Charlotte, North Carolina, 28203, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2578, United States

RECRUITING

Children's Medical Center Dallas

Dallas, Texas, 75235, United States

RECRUITING

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

Houston, Texas, 77030, United States

RECRUITING

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

RECRUITING

Hôpital de la Timone

Marseille, Bouches-du-Rhône, 13385, France

RECRUITING

Angers University Hospital Center

Angers, Maine-et-Loire, 49 933, France

RECRUITING

CHU de Toulouse - Hôpital des Enfants

Toulouse, Occitanie, France

RECRUITING

South Paris University Hospitals - Bicetre Hospital

Le Kremlin-Bicêtre, Paris, 94270, France

RECRUITING

Hôpital Robert-Debré

Paris, Paris, 75019, France

RECRUITING

Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

Florence, Florence, Firenze, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, Genoa, 16147, Italy

NOT YET RECRUITING

Central University Hospital of Asturias

Oviedo, Principality of Asturias, 33011, Spain

NOT YET RECRUITING

MeSH Terms

Conditions

Noonan Syndrome; Turner Syndrome; Dwarfism; Musculoskeletal Diseases; Bone Diseases

Interventions

vosoritide

Condition Hierarchy (Ancestors)

Craniofacial Abnormalities; Musculoskeletal Abnormalities; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases; Bone Diseases, Developmental

Study Officials

• Medical Director, MD, PhD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Central Study Contacts

Trial Specialist

CONTACT

1-800-983-4587; medinfo@bmrn.com

Study Manager

CONTACT

1-800-983-4587; Medinfo@bmrn.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: October 31, 2024
• Study Start: November 22, 2024
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): September 1, 2041
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (5); ES (1); IT (2); AU (1); US (15)