BMN 110 US Expanded Access Program
A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA
1 other identifier
expanded_access
N/A
2 countries
25
Brief Summary
The Expanded Access Program (EAP) is an open-label, multicenter program to:
- 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
Trial Health
Trial Health Score
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25 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 15, 2013
CompletedFirst Posted
Study publicly available on registry
May 21, 2013
CompletedApril 2, 2014
March 1, 2014
May 15, 2013
March 31, 2014
Conditions
Keywords
Interventions
Eligibility Criteria
You may qualify if:
- Diagnosed with MPS IVA as confirmed by either N-acetylgalactosamine-6-sulfatase (GALNS) enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range) or molecular diagnostic test (two mutations in GALNS identified that have previously been associated with an enzyme defect).
- 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.
You may not qualify if:
- Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the program. Patients who become pregnant during the program will be discontinued from the program.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (25)
Unknown Facility
Birmingham, Alabama, 35294, United States
Unknown Facility
Little Rock, Arkansas, 72202, United States
Unknown Facility
Oakland, California, 94609, United States
Unknown Facility
Orange, California, United States
Unknown Facility
Aurora, Colorado, United States
Unknown Facility
Washington D.C., District of Columbia, 20010, United States
Unknown Facility
Hollywood, Florida, 33021, United States
Unknown Facility
Miami, Florida, United States
Unknown Facility
Atlanta, Georgia, 30033, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
Louisville, Kentucky, United States
Unknown Facility
New Orleans, Louisiana, United States
Unknown Facility
Minneapolis, Minnesota, 55404, United States
Unknown Facility
Paterson, New Jersey, United States
Unknown Facility
Manhasset, New York, 11030, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Portland, Oregon, 97239, United States
Unknown Facility
Philadelphia, Pennsylvania, United States
Unknown Facility
Pittsburgh, Pennsylvania, 15224, United States
Unknown Facility
Nashville, Tennessee, United States
Unknown Facility
Houston, Texas, United States
Unknown Facility
Salt Lake City, Utah, 84113, United States
Unknown Facility
Seattle, Washington, United States
Unknown Facility
Tacoma, Washington, 98405, United States
Unknown Facility
Santurce, Puerto Rico