A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With an Inadequate Response to Human Growth Hormone. (CANOPY NS, TS, SHOX-D-2)
Status: Recruiting
Location: See all (20) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 11
Healthy Volunteers: f
View:
• 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 \> -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.
• 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.
• 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.
• Inadequate response to prior hGH treatment.
Locations
United States
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Delaware
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
RECRUITING
Wilmington
Florida
Nicklaus Children's Hospital
NOT_YET_RECRUITING
Miami
Idaho
St. Luke's Children's Endocrinology and Diabetes
RECRUITING
Boise
Kentucky
Kentucky Children's Hospital
RECRUITING
Lexington
Massachusetts
New York Medical College
RECRUITING
Boston
Minnesota
M Health Fairview Pediatric Specialty Clinic - Explorer
RECRUITING
Minneapolis
North Carolina
Atrium Health Carolinas Medical Center
RECRUITING
Charlotte
New York
Icahn School of Medicine at Mount Sinai
RECRUITING
New York
Children's Hospital at Montefiore
RECRUITING
The Bronx
Texas
Children's Medical Center Dallas
RECRUITING
Dallas
University of Texas Health Science Center at Houston (UT Health)
RECRUITING
Houston
Other Locations
France
Angers University Hospital Center
RECRUITING
Angers
South Paris University Hospitals - Bicetre Hospital
RECRUITING
Le Kremlin-bicêtre
Hôpital de la Timone
RECRUITING
Marseille
Hôpital Robert-Debré
RECRUITING
Paris
CHU de Toulouse - Hôpital des Enfants
RECRUITING
Toulouse
Italy
IRCCS Istituto Giannina Gaslini
NOT_YET_RECRUITING
Genova
Spain
Central University Hospital of Asturias
NOT_YET_RECRUITING
Oviedo
Contact Information
Primary
Trial Specialist
medinfo@bmrn.com
1-800-983-4587
Backup
Study Manager
Medinfo@bmrn.com
1-800-983-4587
Time Frame
Start Date:2024-11-22
Estimated Completion Date:2041-09
Participants
Target number of participants:72
Treatments
Experimental: Vosoritide Dose 1 - Low Dose
Drug: Vosoritide - Dose 1 Injection~• Experimental Drug Lyophilized powder for reconstitution
Experimental: Vosoritide Dose 2 - Medium Dose
Drug: Vosoritide - Dose 2 Injection~• Experimental Drug Lyophilized powder for reconstitution
Experimental: Vosoritide Dose 3- High Dose
Drug: Vosoritide Dose 3 Injection~• Experimental Drug Lyophilized powder for reconstitution