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

PHASE3CompletedINTERVENTIONAL
Enrollment

173

Participants

Timeline

Start Date

July 31, 2011

Primary Completion Date

June 16, 2016

Study Completion Date

June 16, 2016

Conditions
Mucopolysaccharidosis IV AMorquio A SyndromeMPS IVA
Interventions
DRUG

BMN 110 - Weekly

"In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week.~In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo."

DRUG

BMN 110 - Every Other Week

"In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks.~In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo."

Trial Locations (43)

Unknown

Phoenix

Oakland

Orange

Wilmington

Washington D.C.

Orlando

Honolulu

Chicago

New York

Seattle

Córdoba

Campina Grande

Porto Alegre

Rio de Janeiro

Montreal

Sherbrooke

Toronto

Bogotá

Copenhagen

Lyon

Marseille

Mainz

Monza

Tokyo

Amsterdam

Oslo

Coimbra

Lisbon

Doha

Riyadh

Seoul

Santiago de Compostela

Taipei

Ankara

Cedex 12

Paris

Cedex 15

Paris

BT9 7AB

Belfast

B15 2TH

Birmingham

B4 6NH

Birmingham

NW3 2PF

London

WC1N 3BG

London

WC1N 3JH

London

M13 9WL

Manchester

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY