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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).
This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| vosoritide injection with vial and syringe | Experimental |
| |
| Placebo injection with vial and syringe | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vosoritide | Drug | Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo | At week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in standing height at Week 52 versus placebo | At week 52 | |
| Change from baseline in height Z-score at Week 52 versus placebo | At week 52 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Alfred I. DuPont Hospital for Children | Wilmington | Delaware | 19803 | United States | ||
| Children's National Medical Center |
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| Placebo | Drug | Subcutaneous injection of recommended dose of placebo |
|
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| University of Alberta Stollery Children's Hospital | Edmonton | Alberta | Canada |
| SickKids - The Hospital for Sick Children | Toronto | Ontario | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | Canada |
| 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 |
| ID | Term |
|---|---|
| C562937 | Hypochondroplasia |
| D004392 | Dwarfism |
| D001847 | Bone Diseases |
| D001848 | Bone Diseases, Developmental |
| D009140 | Musculoskeletal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D010009 | Osteochondrodysplasias |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004700 | Endocrine System Diseases |
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| ID | Term |
|---|---|
| C000632572 | vosoritide |
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