Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

• Status: Completed
• Trial end date: 2016-06-16
• Target enrollment: 0
• Participant gender: All

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 Syndrome).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioMarin Pharmaceutical

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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.