Ignite Creation Date:
2025-12-25 @ 5:02 AM
Ignite Modification Date:
2025-12-26 @ 4:04 AM
Study NCT ID:
NCT03424018
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-01-09
First Post:
2017-12-28
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000130', 'term': 'Achondroplasia'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D004392', 'term': 'Dwarfism'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D010009', 'term': 'Osteochondrodysplasias'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000632572', 'term': 'vosoritide'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 119}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2017-12-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2031-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-07', 'studyFirstSubmitDate': '2017-12-28', 'studyFirstSubmitQcDate': '2018-01-31', 'lastUpdatePostDateStruct': {'date': '2025-01-09', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-02-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2031-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Optional exploratory genomic biomarker analysis', 'timeFrame': 'Once through study completion', 'description': 'Exploratory genomic analysis of genes associated with CNP signaling'}], 'primaryOutcomes': [{'measure': 'Change from baselines in mean annualized growth velocity', 'timeFrame': 'Through study completion, an average of 1 year', 'description': 'Long term efficacy as measured by change in annualized growth velocity'}], 'secondaryOutcomes': [{'measure': 'Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire', 'timeFrame': 'Through study completion, every 6-12 months'}, {'measure': 'Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize maximum concentration (Cmax) of BMN 111 in plasma', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the elimination half-life of BMN 111 (t1⁄2)', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the apparent clearance of drug', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)', 'timeFrame': 'Through study completion, every 12 months'}, {'measure': 'BMN 111 Activity Biomarkers', 'timeFrame': 'Through study completion, every 12 months', 'description': 'BMN 111 activity will be assessed by measuring bone and collagen metabolism'}, {'measure': 'Evaluate change from baseline in body proportion ratios of the extremities', 'timeFrame': 'Through study completion, every 6 months'}, {'measure': 'Effect of BMN 111 on bone morphology and quality', 'timeFrame': 'Through study completion, every 12 months for DXA or 2 years for X-ray', 'description': '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'}, {'measure': 'Final Adult Height', 'timeFrame': 'Up to at least 16 years of age for females and 18 years of age for males', 'description': 'Height at 16 years for females and 18 years for males'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ACH', 'Achondroplasia', 'Bone Diseases', 'Bone Diseases, Developmental', 'Dwarfism', 'Genetic Diseases, Inborn', 'Musculoskeletal Diseases', 'Natriuretic Agents', 'Natriuretic Peptide, C-Type', 'Osteochondrodysplasias', 'Physiological Effects of Drugs', 'Skeletal Dysplasias'], 'conditions': ['Achondroplasia']}, 'referencesModule': {'references': [{'pmid': '39740666', 'type': 'DERIVED', 'citation': '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.'}, {'pmid': '38649657', 'type': 'DERIVED', 'citation': '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.'}], 'seeAlsoLinks': [{'url': 'https://ghr.nlm.nih.gov/condition/achondroplasia', 'label': 'NIH Genetics Home Reference related topics: Achondroplasia'}, {'url': 'https://rarediseases.info.nih.gov/diseases/8173/achondroplasia', 'label': 'Description NIH Genetic and Rare Diseases Information Center resources: Achondroplasia'}, {'url': 'https://clinicaltrials.gov/ct2/info/fdalinks', 'label': 'U.S. FDA Resources'}]}, 'descriptionModule': {'briefSummary': '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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Must have completed Study 111-301\n* Female \\>= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study\n* If sexually active, willing to use a highly effective method of contraception while participating in the study\n* Are willing and able to perform all study procedures\n* Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.\n\nExclusion Criteria:\n\n* Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study\n* Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function\n* Evidence of decreased growth velocity (\\<1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays.\n* Require any investigational agent prior to completion of study period\n* Current therapy with medications known to alter renal function\n* Pregnant or breastfeeding or plan to become pregnant during study\n* Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.\n* Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance or for not completing the study.'}, 'identificationModule': {'nctId': 'NCT03424018', 'briefTitle': 'An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia', 'orgStudyIdInfo': {'id': '111-302'}, 'secondaryIdInfos': [{'id': '2017-002404-28', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'BMN 111', 'interventionNames': ['Drug: BMN 111']}], 'interventions': [{'name': 'BMN 111', 'type': 'DRUG', 'otherNames': ['Vosoritide', 'Modified recombinant human C-type natriuretic peptide'], 'description': 'Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.', 'armGroupLabels': ['BMN 111']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital & Research Center Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '90509', 'city': 'Torrance', 'state': 'California', 'country': 'United States', 'facility': 'Harbor - UCLA Medical Center', 'geoPoint': {'lat': 33.83585, 'lon': -118.34063}}, {'zip': '19803', 'city': 'Wilmington', 'state': 'Delaware', 'country': 'United States', 'facility': 'Alfred I. duPont Hospital for Children', 'geoPoint': {'lat': 39.74595, 'lon': -75.54659}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann and Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '65201', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor College of Medicine', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': "Medical College of Wisconsin, Children's Hospital", 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': "The Children's Hospital at Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': "Murdoch Children's Research Institute", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '39120', 'city': 'Magdeburg', 'country': 'Germany', 'facility': 'Otto-von-Gericke Universitaet, Universitaetskinderklinik', 'geoPoint': {'lat': 52.13129, 'lon': 11.63189}}, {'zip': '48149', 'city': 'Münster', 'country': 'Germany', 'facility': 'Universitätsklinikum Münster', 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'city': 'Osaka', 'country': 'Japan', 'facility': 'Osaka University Hospital', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'city': 'Saitama', 'country': 'Japan', 'facility': "Saitama Children's Medical Center", 'geoPoint': {'lat': 35.90807, 'lon': 139.65657}}, {'city': 'Tokushima', 'country': 'Japan', 'facility': 'Tokushima University Hospital', 'geoPoint': {'lat': 34.06667, 'lon': 134.56667}}, {'zip': '08028', 'city': 'Barcelona', 'country': 'Spain', 'facility': "Institut Catala de Traumatologica I Medicina de l'Esport", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Deu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '29010', 'city': 'Málaga', 'country': 'Spain', 'facility': 'Hospital Universitario Virgen de la Victoria', 'geoPoint': {'lat': 36.72016, 'lon': -4.42034}}, {'zip': '34752', 'city': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Acibadem University School of Medicine', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}, {'zip': 'SE1 9RT', 'city': 'London', 'country': 'United Kingdom', 'facility': "Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'S10 2TH', 'city': 'Sheffield', 'country': 'United Kingdom', 'facility': "Sheffield Children's NHS Foundation Trust", 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}