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

NCT02232477 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-10-31

Study results available
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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.

Conditions

Interventions

DRUG

Intrathecal recombinant human alpha iduronidase

Intrathecal recombinant human alpha iduronidase every 3 months

Sponsors & Collaborators

  • University of Minnesota

    collaborator OTHER

  • UCSF Benioff Children's Hospital Oakland

    collaborator OTHER

  • University of California, Los Angeles

    collaborator OTHER

  • The Ryan Foundation

    collaborator OTHER

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • agnes chen

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-08-31
Primary Completion
2020-02-29
Completion
2020-02-29

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02232477 on ClinicalTrials.gov