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Natural History Studies of Mucopolysaccharidosis III

NCT02037880 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 25

Last updated 2015-11-16

No results posted yet for this study

Summary

The purpose of this study is to assess rates of decline in motor and cognitive functional measures, and to assess potential biomarkers, in order to identify potential outcome measure appropriate for use in therapeutic clinical trials.

Conditions

  • Mucopolysaccharidosis Type IIIA
  • Mucopolysaccharidosis Type IIIB

Interventions

PROCEDURE

Lumbar puncture

DEVICE

Magnetic Resonance Imaging (MRI) of the brain

Sponsors & Collaborators

  • Sanfilippo Children's Research Foundation

    collaborator UNKNOWN

  • The Sanfilippo Research Foundation

    collaborator UNKNOWN

  • The Children's Medical Research Foundation

    collaborator UNKNOWN

  • Nationwide Children's Hospital

    lead OTHER

Principal Investigators

  • Kevin M Flanigan, MD · Nationwide Children's Hospital, Columbus, Ohio

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-28
Primary Completion
2015-11-30
Completion
2015-11-30

Countries

  • United States

Study Locations

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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 NCT02037880 on ClinicalTrials.gov