A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT ID: NCT01275066
Last Updated: 2014-07-07
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
177 participants
INTERVENTIONAL
2011-02-28
2012-08-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes.
This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01242111
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01415427
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01515956
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT01609062
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
NCT00884949
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Placebo
Placebo
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
BMN 110 Weekly
BMN 110 Weekly
BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
BMN 110 Every Other Week
BMN 110 Every Other Week
BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
BMN 110 Weekly
BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Placebo
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
BMN 110 Every Other Week
BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.
* Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
* Must meet the study entrance requirements for the 6-minute walk test.
* Sexually active patients must be willing to use an acceptable method of contraception while participating in the study.
* Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.
Exclusion Criteria
* Previous treatment with BMN 110.
* Has known hypersensitivity to any of the components of BMN 110.
* Major surgery within 3 months prior to study entry or planned major surgery during the 24-week treatment period.
* Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
* Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
* Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
* Any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
5 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
BioMarin Pharmaceutical
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Debra Lounsbury
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Oakland, California, United States
Wilmington, Delaware, United States
Washington D.C., District of Columbia, United States
Chicago, Illinois, United States
New York, New York, United States
Seattle, Washington, United States
Córdoba, , Argentina
Campina Grande, , Brazil
Porto Alegre, , Brazil
Montreal, , Canada
Sherbrooke, , Canada
Toronto, , Canada
Bogotá, , Colombia
Copenhagen, , Denmark
Lyon, , France
Paris, , France
Mainz, , Germany
Monza, , Italy
Tokyo, , Japan
Amsterdam, , Netherlands
Coimbra, , Portugal
Doha, , Qatar
Riyadh, , Saudi Arabia
Seoul, , South Korea
Taipei, , Taiwan
Birmingham, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.
Related Links
Access external resources that provide additional context or updates about the study.
BioMarin Pharmaceutical Inc. website
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2010-020198-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
10/H1306/87
Identifier Type: OTHER
Identifier Source: secondary_id
18972/0213/001-0001
Identifier Type: OTHER
Identifier Source: secondary_id
2011_038#B201129
Identifier Type: OTHER
Identifier Source: secondary_id
145240
Identifier Type: OTHER
Identifier Source: secondary_id
2011-01-09
Identifier Type: OTHER
Identifier Source: secondary_id
20110012889
Identifier Type: OTHER
Identifier Source: secondary_id
0999935174
Identifier Type: OTHER
Identifier Source: secondary_id
MOR-004
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.