NCT06668805
Noonan Syndrome
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 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.
All
3 Years to 11 Years
No
Vosoritide Injection
Phase 2
Interventional
30
2024-11-22
2026-05-11
Los Angeles, California, United States
Trial Status Recruiting
Anna Ryabets-Lienhard
323-361-3861 jobarajas@chla.usc.edu
Orange, California, United States
Trial Status Recruiting
Amrit Bhangoo
7145098634 abhangoo@choc.org
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
Atlanta, Georgia, United States
Trial Status Recruiting
William Wilcox
(404) 778-8518 william.wilcox@emory.edu
Boise, Idaho, United States
Trial Status Recruiting
Daniel Flynn
2083817340 flynnd@slhs.org
Chicago, Illinois, United States
Trial Status Recruiting
Carlos Prada
312-227-6120
Indianapolis, Indiana, United States
Trial Status Recruiting
John Fuqua
3172741201 jsfuqua@iu.edu
Iowa City, Iowa, United States
Trial Status Recruiting
Michael Tansey
(319) 356-4443
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
Cincinnati, Ohio, United States
Trial Status Recruiting
Gajanthan Muthuvel
(513) 636-8444
Philadelphia, Pennsylvania, United States
Trial Status Recruiting
Vaneeta Bamba
215-590-4470 paris@chop.edu
Nashville, Tennessee, United States
Trial Status Recruiting
Scott Ward
615-936-5475 scott.k.ward@vumc.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
Saint Leonards, New South Wales, Australia
Trial Status Recruiting
Shihab Hameed
61294631127 NSLHD-CTPharmacy@health.nsw.gov.au
South Brisbane, Queensland, Australia
Trial Status Recruiting
Tony Huynh
61730681951 Anita.Champion@health.qld.gov.au
Parkville, Victoria, Australia
Trial Status Recruiting
Ravi Savarirayan
+61399366541 ravi.savarirayan@mcri.edu.au
Calgary, Alberta, Canada
Trial Status Recruiting
Aneal Khan
5878853158 april.williams@magiccalgary.ca
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
Homburg, Saarland, Germany
Trial Status Recruiting
Tilman Rohrer
+49 (0) 68-41-16-2-83-01
Florence, Florence, Italy
Trial Status Recruiting
Stefano Stagi
+39 3488421557 stefano.stagi@unifi.it
Genova, Genoa, Italy
Trial Status Not yet recruiting
Mohamad Maghnie
mohamadmaghnie@gaslini.org
Roma, Italy
Trial Status Recruiting
Giuseppe Zampino
06 30156050 farmaciasperimentali@policlinicogemelli.it
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 ≤ -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
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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