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COMPLETEDNCT02950298

Pompe Telemedicine Developmental Study

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)...

Lead sponsor: Duke University•1 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Duke University

The study will last approximately 2-3 years. It includes 2-4 visits to Duke for developmental assessments. The baseline/year 1 visit may take place at Duke (may take 1-2 days) or remotely via iPad. The follow up 1/year 2 visit may take place at Duke (1-2 days) or remotely via an iPad, depending upon each child's particular situation.

Age

6 - 18 years

Sex

ALL

Healthy Volunteers

No

Inclusion Criteria

•Age range 6-18 years
•Diagnosis of classic Pompe disease by enzyme or molecular methods
•Patient, parent, or legal guardian is willing and able to give written informed consent
•English speaking child and care giver.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: October 28, 2021Terms

Trial snapshot

StatusCOMPLETED

Phase—

Enrollment22

Start dateDec 2015

Last updatedOct 28, 2021

Condition

Pompe Disease Glycogen Storage Disease II

Locations shown

Duke University Medical Center

Durham, North Carolina

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 28, 2021Data synced to Clareo: May 10, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions