A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone
NCT ID: NCT06668805
Last Updated: 2025-08-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
72 participants
INTERVENTIONAL
2024-11-22
2041-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Vosoritide Dose 1 - Low Dose
Drug: Vosoritide - Dose 1 Injection
• Experimental Drug Lyophilized powder for reconstitution
Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Vosoritide Dose 2 - Medium Dose
Drug: Vosoritide - Dose 2 Injection
• Experimental Drug Lyophilized powder for reconstitution
Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Vosoritide Dose 3- High Dose
Drug: Vosoritide Dose 3 Injection
• Experimental Drug Lyophilized powder for reconstitution
Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Human Growth Hormone
Drug: Human Growth Hormone
Human Growth Hormone
Commercial product containing somatotropin.
Interventions
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Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Human Growth Hormone
Commercial product containing somatotropin.
Eligibility Criteria
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Inclusion Criteria
2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
3. A height assessment corresponding to a height Z-score of \> -2.00 SDs and ≤ -1.75 SDs (up to 20% of participants)/≤ -2.00 SDs (at least 80% of participants) in reference to the general population of the same age and sex.
4. Tanner Stage 1, at time of signing the ICF.
5. Have been receiving continuous hGH for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment and be receiving a dose of ≥ 0.35 mg/kg weekly, with no weight-based dosing changes in the last 6 months and none planned in the future.
6. Are willing to continue on hGH at their current dose for the Baseline Growth Phase, and for 2 years post randomization if randomized to the hGH arm.
7. Inadequate response to prior hGH treatment.
Exclusion Criteria
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.
3 Years
11 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD, PhD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
Children's Medical Center Dallas
Dallas, Texas, United States
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas, United States
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
IRCCS Istituto Giannina Gaslini
Genova, Genoa, Italy
Central University Hospital of Asturias
Oviedo, Principality of Asturias, Spain
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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111-211
Identifier Type: -
Identifier Source: org_study_id
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