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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-004004-30 | EudraCT Number |
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This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMN 111 - Subcutaneous Injection | Experimental | 111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BMN 111 | Drug | BMN 111 will be administered subcutaneously daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] |
| Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later |
| Measure | Description | Time Frame |
|---|---|---|
| Growth Velocity [Efficacy] | Annualized growth velocity (cm/yr) | Until near final adult height is reached |
| Growth Parameters (Efficacy) | Height standard score (Z-score) |
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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 |
|---|---|---|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | 94609 | United States | ||
| Harbor - UCLA Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 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. | |
| 31269546 |
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| Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later |
| Body Proportions (Efficacy) | Upper-to-lower body segment ratio | Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later |
| Final Adult Height | Height at the age of 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 |
| Ann and Robert H. Lurie Childrens Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Johns Hopkins McKusick - Institute of Genetic Medicine | Baltimore | Maryland | 21287 | United States |
| Vanderbilt University | Nashville | Tennessee | 37232-2578 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| Institut Necker | Paris | 75015 | France |
| Guys & St. Thomas NHS Foundation Trust Evelina Hospital | London | SE1 9RT | United Kingdom |
| Derived |
| Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18. |
| ID | Term |
|---|---|
| D000130 | Achondroplasia |
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| ID | Term |
|---|---|
| D009140 | Musculoskeletal Diseases |
| D010009 | Osteochondrodysplasias |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004700 | Endocrine System Diseases |
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| ID | Term |
|---|---|
| C000632572 | vosoritide |
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