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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

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Trial Status Recruiting

Trial Identifier

NCT06668805

Condition

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

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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 versus hGH 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.

Eligibility Criteria

Birth Sex

All

Age

3 Years to 11 Years

Healthy Volunteers

No

Drug/Treatment:

Vosoritide Injection, Human Growth Hormone

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

72

Study Started:

2024-11-22

Study Updated:

2025-08-06

Trial Locations

  • Children's Hospital Colorado

    Aurora, Colorado, United States

    Trial Status Recruiting

    Contact

    Shanlee Davis
    720-777-5251 shanlee.davis@childrenscolorado.org

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

    Wilmington, Delaware, United States

    Trial Status Recruiting

    Contact

    Chijioke Ikomi
    302-651-5965 chijioke.ikomi@nemours.org

  • Children's National Medical Center

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

    Trial Status Recruiting

    Contact

    Andrew Dauber
    adauber@childrensnational.org

  • Nicklaus Children's Hospital

    Miami, Florida, United States

    Trial Status Not yet recruiting

    Contact

    Ana Maria Rodriguez Barreto
    anamaria.rodriguezbarreto@nicklaushealth.org

  • St. Luke's Children's Endocrinology and Diabetes

    Boise, Idaho, United States

    Trial Status Recruiting

    Contact

    Daniel Flynn
    2083817340 flynnd@slhs.org

  • Kentucky Children's Hospital

    Lexington, Kentucky, United States

    Trial Status Recruiting

    Contact

    Yuri Zarate
    859-257-1000 yuri.zarate@uky.edu

  • New York Medical College

    Boston, Massachusetts, United States

    Trial Status Recruiting

    Contact

    Shilpa Mehta
    shilpa_mehta@bchphysicians.org

  • M Health Fairview Pediatric Specialty Clinic - Explorer

    Minneapolis, Minnesota, United States

    Trial Status Recruiting

    Contact

    Kyriakie Sarafoglou
    saraf010@umn.edu

  • Icahn School of Medicine at Mount Sinai

    New York, New York, United States

    Trial Status Recruiting

    Contact

    Cassie Mintz
    cassie.mintz@mssm.edu

  • Children's Hospital at Montefiore

    The Bronx, New York, United States

    Trial Status Recruiting

    Contact

    Laurie Cohen
    718-920-4664 lacohen@montefiore.org

  • Atrium Health Carolinas Medical Center

    Charlotte, North Carolina, United States

    Trial Status Recruiting

    Contact

    Edwin Ferren
    edwin.ferren@atriumhealth.org

  • Children's Medical Center Dallas

    Dallas, Texas, United States

    Trial Status Recruiting

    Contact

    Nadia Merchant
    214-456-5959 nadia.merchant@utsouthwestern.edu

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

    Houston, Texas, United States

    Trial Status Recruiting

    Contact

    David Rodriguez Buritica
    david.f.rodriguezburitica@uth.tmc.edu

  • Hôpital de la Timone

    Marseille, Bouches-du-Rhône, France

    Trial Status Recruiting

    Contact

    Rachel Reynaud
    rachel.reynaud@ap-hm.fr

  • Angers University Hospital Center

    Angers, Maine-et-Loire, France

    Trial Status Recruiting

    Contact

    Regis Coutant
    recoutant@chu-angers.fr

  • CHU de Toulouse - Hôpital des Enfants

    Toulouse, Occitanie, France

    Trial Status Recruiting

    Contact

    Thomas Edouard
    edouard.t@chu-toulouse.fr

  • South Paris University Hospitals - Bicetre Hospital

    Le Kremlin-Bicêtre, Paris, France

    Trial Status Recruiting

    Contact

    Agnes Linglart
    agnes.linglart@aphp.fr

  • Hôpital Robert-Debré

    Paris, Paris, France

    Trial Status Recruiting

    Contact

    Jean-Claude Carel
    jean-claude.carel@rdb.aphp.fr

  • IRCCS Istituto Giannina Gaslini

    Genova, Genoa, Italy

    Trial Status Not yet recruiting

    Contact

    Mohamad Maghnie
    mohamadmaghnie@gaslini.org

  • Central University Hospital of Asturias

    Oviedo, Principality of Asturias, Spain

    Trial Status Not yet recruiting

    Contact

    Isolina Riano Galan
    isolinariano@gmail.com

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 > -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.
    Tanner Stage 1, at time of signing the ICF.
  4. 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.
  5. 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.
  6. Inadequate response to prior hGH treatment.

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.

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