NCT06668805
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.
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.
All
3 Years to 11 Years
No
Vosoritide Injection, Human Growth Hormone
Phase 2
Interventional
72
2024-11-22
2025-08-06
Aurora, Colorado, United States
Trial Status Recruiting
Shanlee Davis
720-777-5251 shanlee.davis@childrenscolorado.org
Wilmington, Delaware, United States
Trial Status Recruiting
Chijioke Ikomi
302-651-5965 chijioke.ikomi@nemours.org
Washington D.C., District of Columbia, United States
Trial Status Recruiting
Andrew Dauber
adauber@childrensnational.org
Miami, Florida, United States
Trial Status Not yet recruiting
Ana Maria Rodriguez Barreto
anamaria.rodriguezbarreto@nicklaushealth.org
Boise, Idaho, United States
Trial Status Recruiting
Daniel Flynn
2083817340 flynnd@slhs.org
Lexington, Kentucky, United States
Trial Status Recruiting
Yuri Zarate
859-257-1000 yuri.zarate@uky.edu
Boston, Massachusetts, United States
Trial Status Recruiting
Shilpa Mehta
shilpa_mehta@bchphysicians.org
Minneapolis, Minnesota, United States
Trial Status Recruiting
Kyriakie Sarafoglou
saraf010@umn.edu
New York, New York, United States
Trial Status Recruiting
Cassie Mintz
cassie.mintz@mssm.edu
The Bronx, New York, United States
Trial Status Recruiting
Laurie Cohen
718-920-4664 lacohen@montefiore.org
Charlotte, North Carolina, United States
Trial Status Recruiting
Edwin Ferren
edwin.ferren@atriumhealth.org
Dallas, Texas, United States
Trial Status Recruiting
Nadia Merchant
214-456-5959 nadia.merchant@utsouthwestern.edu
Houston, Texas, United States
Trial Status Recruiting
David Rodriguez Buritica
david.f.rodriguezburitica@uth.tmc.edu
Marseille, Bouches-du-Rhône, France
Trial Status Recruiting
Rachel Reynaud
rachel.reynaud@ap-hm.fr
Angers, Maine-et-Loire, France
Trial Status Recruiting
Regis Coutant
recoutant@chu-angers.fr
Toulouse, Occitanie, France
Trial Status Recruiting
Thomas Edouard
edouard.t@chu-toulouse.fr
Le Kremlin-Bicêtre, Paris, France
Trial Status Recruiting
Agnes Linglart
agnes.linglart@aphp.fr
Paris, Paris, France
Trial Status Recruiting
Jean-Claude Carel
jean-claude.carel@rdb.aphp.fr
Genova, Genoa, Italy
Trial Status Not yet recruiting
Mohamad Maghnie
mohamadmaghnie@gaslini.org
Oviedo, Principality of Asturias, Spain
Trial Status Not yet recruiting
Isolina Riano Galan
isolinariano@gmail.com
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.
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.
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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