Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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APPROVED_FOR_MARKETING
EXPANDED_ACCESS
Brief Summary
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1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available
2. Collect additional information on the safety and tolerability of BMN 110 administration in patients with MPS IVA
Patients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110 every week during the program.
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Detailed Description
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Conditions
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Interventions
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BMN 110
Eligibility Criteria
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Inclusion Criteria
* Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18, provide written assent (as required by the IRB) and written informed consent by a legally authorized representative after the nature of the program has been explained, and prior to any program assessments or evaluations.
* Sexually active patients must be willing to use an acceptable method of contraception while participating in the program.
* Females of childbearing potential must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the program.
* Willing and able to comply with all program procedures.
Exclusion Criteria
* Currently enrolled in an ongoing clinical study of BMN 110.
* Discontinued from a BMN 110 clinical study secondary to a safety-related event.
* Use of any investigational product (other than BMN 110 in a clinical study) or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled program assessments.
* Not a current US resident or expecting to have travel plans outside the US during the planned period of participation in the Expanded Access Program (EAP) that may interfere with dosing regimen, scheduled program visits and safety monitoring.
* Any condition that, in the view of the Investigator or sponsor, places the patient at high risk of poor treatment compliance or of not completing the EAP.
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Locations
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Birmingham, Alabama, United States
Little Rock, Arkansas, United States
Oakland, California, United States
Orange, California, United States
Aurora, Colorado, United States
Washington D.C., District of Columbia, United States
Hollywood, Florida, United States
Miami, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Louisville, Kentucky, United States
New Orleans, Louisiana, United States
Minneapolis, Minnesota, United States
Paterson, New Jersey, United States
Manhasset, New York, United States
New York, New York, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Nashville, Tennessee, United States
Houston, Texas, United States
Salt Lake City, Utah, United States
Seattle, Washington, United States
Tacoma, Washington, United States
Santurce, , Puerto Rico
Countries
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Other Identifiers
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110-503
Identifier Type: -
Identifier Source: org_study_id
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