Clinical Trials in Italy / NCT06455059
Active, not recruiting
Phase 3
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT06455059 · tracked via the Priya Life Science Italy tracker
Condition(s) studied
Hypochondroplasia
Investigational drug(s) / intervention(s)
Vosoritide →Placebo
Vosoritide: Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Placebo: Subcutaneous injection of recommended dose of placebo
Study summary
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
Eligibility
Inclusion Criteria:
1. Participants must be ≥ 3 to \< 18 years of age at enrollment
2. A confirmed genetic diagnosis of HCH
3. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts
4. Males and females are eligible to participate in this clinical study.
5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
Exclusion Criteria:
1. Short stature condition other than HCH
2. Have an unstable condition likely to require surgical intervention during the study.
3. Evidence of decreased growth velocity and/or growth plate closure
4. Taking any of the prohibited medications
5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
6. Planned or expected to have limb-lengthening surgery during the study period.
7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
8. Require any investigational agent prior to completion of study period.
9. Received vosoritide or another investigational product or investigational medical device in the past
10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
12. Have known hypersensitivity to vosoritide or its excipients.
13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Primary outcome measure(s)
- Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo — At week 52
Trial sites (23)
| Facility | City | Region | Status |
|---|---|---|---|
| Nemours Alfred I. DuPont Hospital for Children | Wilmington | Delaware | |
| Children's National Medical Center | Washington D.C. | District of Columbia | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | |
| Medical College of Wisconsin | Milwaukee | Wisconsin | |
| Murdoch Children's Research Institute | Parkville | Victoria | |
| University of Alberta Stollery Children's Hospital | Edmonton | Alberta | |
| SickKids - The Hospital for Sick Children | Toronto | Ontario | |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | |
| Hospices Civils de Lyon - Hopital Femme Mere Enfant | Lyon | France | |
| Hopital de la Timone | Marseille | France | |
| Hopital Necker-Enfants Malade | Paris | France | |
| CHU de Toulouse | Toulouse | France | |
| Uniklinik Koln | Cologne | Germany | |
| Univeristatskinderklinik Magdeburg | Magdeburg | Germany | |
| Instituto Giannina Gaslini | Genoa | Italy | |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Rome | Italy | |
| Ospedale Pediatrico Bambino Gesu | Rome | Italy | |
| Osaka University Hosptial | Osaka | Japan | |
| Tokushima University Hospital | Tokushima | Japan | |
| Institute of Science Tokyo Hospital | Tokyo | Japan | |
| Tottori University Hospital | Tottori | Japan | |
| Vithas Hospital San Jose | Vitoria-Gasteiz | Spain | |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06455059 on ClinicalTrials.gov ↗ ← All trials in Italy