Clinical Trial: Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I

Brief Summary: This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study.

Detailed Summary:
Sponsor: agnes chen

Current Primary Outcome: Hopkins Verbal Learning Test [ Time Frame: 5 years ]

Mean intra-subject change in memory score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Los Angeles Biomedical Research Institute

Dates:
Date Received: September 3, 2014
Date Started: May 2014
Date Completion: June 2020
Last Updated: May 5, 2017
Last Verified: May 2017