Intrathecal Enzyme Replacement for Hurler Syndrome

PHASE1CompletedINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

January 2, 2008

Primary Completion Date

February 18, 2017

Study Completion Date

November 18, 2018

Conditions
Hurler Syndrome
Interventions
DRUG

IRT Laronidase

"Laronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup.~Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture"

Trial Locations (1)

55455

University of Minnesota, Fairview, Minneapolis

All Listed Sponsors
lead

Masonic Cancer Center, University of Minnesota

OTHER

NCT00638547 - Intrathecal Enzyme Replacement for Hurler Syndrome | Biotech Hunter | Biotech Hunter