Status:
COMPLETED
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Mucopolysaccharidosis IV A
Morquio A Syndrome
Eligibility:
All Genders
5+ years
Phase:
PHASE3
Brief Summary
This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A S...
Detailed Description
This is a multi-center, multinational, extension study to evaluate 2 dose regimens of BMN 110 treatment in patients with MPS IVA who completed MOR-004. The last study visit assessments for MOR-004 wi...
Eligibility Criteria
Inclusion
- Must have completed MOR-004
- Is willing and able to provide written, signed informed consent. Or in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorize representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
- If sexually active, must be willing to use an acceptable method of contraception while participating in the study.
- If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study.
Exclusion
- Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study.
- Has used any investigational product (other than BMN 110 in MOR-004), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
- Was enrolled in a previous BMN 110 study, other than MOR-004.
- Has a 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 pose a safety risk, as determined by the Investigator.
- Has 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.
Key Trial Info
Start Date :
July 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 16 2016
Estimated Enrollment :
173 Patients enrolled
Trial Details
Trial ID
NCT01415427
Start Date
July 1 2011
End Date
June 16 2016
Last Update
July 21 2021
Active Locations (43)
Enter a location and click search to find clinical trials sorted by distance.
1
Phoenix, Arizona, United States
2
Oakland, California, United States
3
Orange, California, United States
4
Wilmington, Delaware, United States