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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002404-28 | EudraCT Number |
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The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMN 111 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BMN 111 | Drug | Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from baselines in mean annualized growth velocity | Long term efficacy as measured by change in annualized growth velocity | Through study completion, an average of 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire | Through study completion, every 6-12 months | |
| Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire |
| Measure | Description | Time Frame |
|---|---|---|
| Optional exploratory genomic biomarker analysis | Exploratory genomic analysis of genes associated with CNP signaling | Once through study completion |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | 94609 | United States | ||
| Harbor - UCLA Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39740666 | Derived | Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025 May 9;6(5):100566. doi: 10.1016/j.medj.2024.11.019. Epub 2024 Dec 30. | |
| 39305160 |
| Label | URL |
|---|---|
| NIH Genetics Home Reference related topics: Achondroplasia | View source |
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| Through study completion, every 12 months |
| Characterize maximum concentration (Cmax) of BMN 111 in plasma | Through study completion, every 12 months |
| Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞) | Through study completion, every 12 months |
| Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t) | Through study completion, every 12 months |
| Characterize the elimination half-life of BMN 111 (t1⁄2) | Through study completion, every 12 months |
| Characterize the apparent clearance of drug | Through study completion, every 12 months |
| Characterize the apparent volume of distribution based upon the terminal phase (Vz/F) | Through study completion, every 12 months |
| Characterize the amount of time BMN 111 is present at maximum concentration (Tmax) | Through study completion, every 12 months |
| BMN 111 Activity Biomarkers | BMN 111 activity will be assessed by measuring bone and collagen metabolism | Through study completion, every 12 months |
| Evaluate change from baseline in body proportion ratios of the extremities | Through study completion, every 6 months |
| Effect of BMN 111 on bone morphology and quality | The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via X-Ray and Dual X-ray Absorptiometry | Through study completion, every 12 months for DXA or 2 years for X-ray |
| Final Adult Height | Height at 16 years for females and 18 years for males | Up to at least 16 years of age for females and 18 years of age for males |
| Torrance |
| California |
| 90509 |
| United States |
| Alfred I. duPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| University of Missouri | Columbia | Missouri | 65201 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Medical College of Wisconsin, Children's Hospital | Milwaukee | Wisconsin | 53226 | United States |
| The Children's Hospital at Westmead | Westmead | New South Wales | 2145 | Australia |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| Otto-von-Gericke Universitaet, Universitaetskinderklinik | Magdeburg | 39120 | Germany |
| Universitätsklinikum Münster | Münster | 48149 | Germany |
| Osaka University Hospital | Osaka | Japan |
| Saitama Children's Medical Center | Saitama | Japan |
| Tokushima University Hospital | Tokushima | Japan |
| Institut Catala de Traumatologica I Medicina de l'Esport | Barcelona | 08028 | Spain |
| Hospital Sant Joan de Deu | Barcelona | 08950 | Spain |
| Hospital Universitario Virgen de la Victoria | Málaga | 29010 | Spain |
| Acibadem University School of Medicine | Istanbul | 34752 | Turkey (Türkiye) |
| Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital | London | SE1 9RT | United Kingdom |
| Sheffield Children's NHS Foundation Trust | Sheffield | S10 2TH | United Kingdom |
| Derived |
| Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A, Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J. Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life. Genet Med. 2024 Dec;26(12):101274. doi: 10.1016/j.gim.2024.101274. Epub 2024 Sep 18. |
| 38649657 | Derived | Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23. |
| Description NIH Genetic and Rare Diseases Information Center resources: Achondroplasia | View source |
| U.S. FDA Resources | View source |
| ID | Term |
|---|---|
| D000130 | Achondroplasia |
| D001847 | Bone Diseases |
| D001848 | Bone Diseases, Developmental |
| D004392 | Dwarfism |
| D030342 | Genetic Diseases, Inborn |
| D009140 | Musculoskeletal Diseases |
| 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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