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Pompe Telemedicine Developmental Study

NCT02950298 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 22

Last updated 2021-10-28

No results posted yet for this study

Summary

The primary purpose of this study is to:

* Document the developmental outcomes of individuals with Pompe disease treated with long-term enzyme-replacement therapy (ERT) through school-age (ages 6-18) using measures of cognitive functioning, academic skills, and speech and language abilities.
* Investigate possible cognitive processing speed weaknesses using BrainBaseline neurocognitive assessment software.
* Investigate the relationship between behavior and other developmental factors including speech and language ability and cognitive ability.
* Explore if the use of selected iPad applications may help strengthen cognitive processing speed in children with Pompe disease

Conditions

  • Pompe Disease
  • Glycogen Storage Disease II

Sponsors & Collaborators

Principal Investigators

  • Priya Kishnani · Duke University

Eligibility

Min Age
6 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-31
Primary Completion
2021-03-31
Completion
2021-03-03

Countries

  • United States

Study Locations

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Entities

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 NCT02950298 on ClinicalTrials.gov