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

Completed · Phase 3 · Has a placebo group

Conditions studied: Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA

In brief

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).

Key facts

Study ID

NCT01415427

Run by

BioMarin Pharmaceutical

People needed

173

Starts

2011-07-01

Expected to finish

2016-06-16

Last updated by the study team

2021-07-21

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Study site — Phoenix, Arizona, United States
• Study site — Oakland, California, United States
• Study site — Orange, California, United States
• Study site — Wilmington, Delaware, United States
• Study site — Washington D.C., District of Columbia, United States
• Study site — Orlando, Florida, United States
• Study site — Honolulu, Hawaii, United States
• Study site — Chicago, Illinois, United States
• Study site — New York, New York, United States
• Study site — Seattle, Washington, United States
• Study site — Córdoba, Argentina
• Study site — Campina Grande, Brazil
• Study site — Porto Alegre, Brazil
• Study site — Rio de Janeiro, Brazil
• Study site — Montreal, Canada
• Study site — Sherbrooke, Canada
• Study site — Toronto, Canada
• Study site — Bogotá, Colombia
• Study site — Copenhagen, Denmark
• Study site — Lyon, France
• Study site — Marseille, France
• Study site — Paris, France
• Study site — Paris, France
• Study site — Mainz, Germany
• Study site — Monza, Italy

Full record on ClinicalTrials.gov

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