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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000701-21 | EudraCT Number |
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Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin. No study drug is administered.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Growth measurements | Approximately 500 patients will be enrolled. Patients from birth to <= 17 years on the date of consent will be enrolled. Approximately equal numbers of boys and girls will be enrolled. |
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| Measure | Description | Time Frame |
|---|---|---|
| Collection of consistent growth measurements | Patients will be screened and undergo a series of growth measurements on Day 1 and every 3 months thereafter. No study drug is administered. | Assessed every 3 months for up to 84 months |
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Inclusion Criteria:
Exclusion Criteria:
Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia)
Have any of the following disorders:
Have an unstable clinical condition likely to lead to intervention during the course of the study, including progressive cervical medullary compression
Growth plates have fused
Have a history of any of the following:
Cardiac or vascular disease, including the following:
Current treatment with antihypertensive medications angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, drugs known to alter renal function that is expected to continue for the duration of the study
Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time
Have had regular long-term treatment (> 1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
Concomitant medication that prolongs the QT/QTc interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit
Have used any other investigational product or investigational medical device for the treatment of ACH or short stature
Have had bone-related surgery or expected to have bone-related surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.
Have any condition that, in the view of the Investigator, places the patient at high risk of poor compliance with the visit schedule or of not completing the study.
Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation
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Approximately 500 patients will be enrolled. Patients from birth to <= 17 years, inclusive, on the date of consent will be enrolled. Approximately equal numbers of boys and girls will be enrolled.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Harbor-UCLA Medical Center | Los Angeles | California | 90048 | United States | ||
| Children's Hospital and Research Center Oakland |
| 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. | |
| 36107167 |
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| Oakland |
| California |
| 94609 |
| United States |
| Nemours/Alfred I. duPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Emory University | Decatur | Georgia | 30033 | United States |
| Ann and Robert H Lurie Children's Hospital of Chicago | Chicago | Illinois | 60614 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Vanderbilt University | Nashville | Tennessee | 37232 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Medical College of Wisconsin, Children's Hospital | Milwaukee | Wisconsin | 53226 | United States |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| The Children's Hospital at Westmead | Westmead | 2145 | Australia |
| 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 | 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, Harmatz P, Delgado B, Wilcox WR, Philips J, Owen N, Bacino CA, Tofts L, Charrow J, Polgreen LE, Hoover-Fong J, Arundel P, Ginebreda I, Saal HM, Basel D, Font RU, Ozono K, Bober MB, Cormier-Daire V, Le Quan Sang KH, Baujat G, Alanay Y, Rutsch F, Hoernschemeyer D, Mohnike K, Mochizuki H, Tajima A, Kotani Y, Weaver DD, White KK, Army C, Larrimore K, Gregg K, Jeha G, Milligan C, Fisheleva E, Huntsman-Labed A, Day J. Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study. Genet Med. 2022 Dec;24(12):2444-2452. doi: 10.1016/j.gim.2022.08.015. Epub 2022 Sep 16. |
| 34431071 | Derived | Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J. Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Clin Pharmacokinet. 2022 Feb;61(2):263-280. doi: 10.1007/s40262-021-01059-1. Epub 2021 Aug 25. |
| 31269546 | 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 |
| ID | Term |
|---|---|
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| 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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