Ignite Creation Date:
2025-12-24 @ 7:48 PM
Ignite Modification Date:
2025-12-30 @ 5:31 AM
Study NCT ID:
NCT01858103
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2014-04-02
First Post:
2013-05-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
BMN 110 US Expanded Access Program
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2014-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
Patients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110 every week during the program.
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.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: