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The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vosoritide | Experimental | Open-label active drug |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vosoritide | Drug | Open-label administration of vosoritide using weight-band dosing |
|
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) | As measured by change from baseline in height Z-score | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the maintenance effect of vosoritide treatment on standing height | change from baseline in height at Week 52 | Baseline to week 52 |
| Evaluate the maintenance effect of vosoritide treatment on standing height Z-score |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) | Wilmington | Delaware | 19803 | United States | ||
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Change from baseline in height Z-score at Week 52
| Baseline to week 52 |
| Evaluate long-term effect of vosoritide treatment on growth velocity | Absolute 6- or 12-month interval AGV | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term effect of vosoritide treatment on body proportionality | Change from baseline in upper to lower body segment ratio | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term effect of vosoritide treatment on bone age versus chronological age | As measured by left-hand and wrist x-ray | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers | Change from baseline in serum collagen X marker | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term changes in Health-Related Quality of Life (HRQoL) | Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term effect of vosoritide treatment on bone quality | Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| To monitor the incidence of otitis media | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| To monitor the frequency of seizures | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
| Children's National Medical Center |
| 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 |
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| ID | Term |
|---|---|
| C000632572 | vosoritide |
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