A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Vosoritide Injection, Human Growth Hormone, Placebo.
- Who it may be relevant to
- Registry conditions: Idiopathic Short Stature. Basic parameters: 3 years — 11 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, France, Germany, Italy +1
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
Overview
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Detailed description
Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.
A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection.
Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee
An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals
Interventions
- Drug Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution - Drug Human Growth Hormone
Commercial product containing somatotropin - Drug Placebo
Lyophilized powder for reconstitution
Primary outcome measures
- Change from baseline in Annualized Growth Velocity (AGV) [Time frame: At 6 months]
- Change from baseline in height [Time frame: At 4 years]
- Change from baseline in height Z-score [Time frame: At 4 years]
Secondary outcome measures (12)
- Incidence of treatment-emergent adverse events [Time frame: Until the end of the study, up to 15 years]
- Change from baseline in Height Z-score (average stature reference) [Time frame: At 6 months]
- Change from baseline in height [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in height Z score [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP) [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline at pre-specified timepoints in serum collagen X marker (CXM) [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in bone age minus chronological age at pre-specified timepoints [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in whole body (less head) bone mineral density (BMD) Z-score [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in lumbar spine BMD Z-score [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in total body (less head) bone mineral content (BMC) [Time frame: Every 6 months through the end of study, up to 15 years]
- Change from baseline in lumbar spine BMC [Time frame: Every 6 months through the end of study, up to 15 years]
- Maximum concentration (Cmax) of vosoritide in plasma [Time frame: Every 6 months through the end of study, up to 15 years]
Eligibility criteria
Inclusion criteria
- Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
- If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).
Key Exclusions:
- Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.
- Previous treatment with a growth promoting agent
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 35 centers
- Consano Clinical Research, LLC — California City
- Rady Children's Hospital - San Diego — California City
- Children's Hospital of Orange County Main Campus - Orange — California City
- Children's Hospital Los Angeles — Los Angeles
- Children's Hospital Oakland Research Institute — Oakland
- Center Of Excellence in Diabetes and Endocrinology — Sacramento
- Lundquist Institute for Biomedical Innovation (LA BioMed) — Torrance
- The Children's National Research Institute — Washington D.C.
- … and 27 more centers
France · 4 centers
- Centre Hospitalier Universitaire d'Angers — Angers
- Hôpital de la Timone - Enfants — Marseille
- CHU de Toulouse - Hôpital des Enfants — Toulouse
- Hôpital Bicêtre — Le Kremlin-Bicêtre
Italy · 3 centers
- Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer — Florence
- Ospedale Pediatrico Bambino Gesù — Roma
- IRCCS Istituto Giannina Gaslini — Genova
South Korea · 3 centers
- Seoul National University Bundang Hospital — Seongnam-si
- Inha University Hospital — Incheon
- Chungnam National University Sejong Hospital (CNUSH) — Sejong
Australia · 1 center
- Murdoch Children's Research Institute — Parkville
Germany · 1 center
- Universitätsklinikum des Saarlandes — Homburg
Identifiers
NCT: NCT06382155 · 111-210