Treatment for Late-Onset Pompe Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Duke University Medical Center, Durham, NC
Late-Onset Pompe Disease+2 More
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial assesses the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and its effects on respiratory and patient-reported outcomes. This trial is testing a new treatment for Late-Onset Pompe Disease. The treatment will be done online. All patients in this trial will receive the new treatment, not a placebo.

Eligible Conditions

  • Late-Onset Pompe Disease
  • Lysosomal Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Late-Onset Pompe Disease

Study Objectives

1 Primary · 22 Secondary · Reporting Duration: Baseline, week 15, week 30

Week 30
Change in ability to communicate
Change in compression phase duration (CPD)
Change in cough volume acceleration (CVA)
Change in daytime sleepiness
Change in expiratory phase rise time (EPRT)
Change in fatigue
Change in fatigue index test score (FIT)
Change in forced expiratory volume over 1 second (FEV1)
Change in forced vital capacity (FVC)
Change in health-related quality of life
Change in impact of fatigue on quality of life (QOL)
Change in inspiratory duration (ID)
Change in inspiratory peak flow (IPF)
Change in inspiratory phase duration (IPD)
Change in inspiratory power curve (IPC)
Change in maximum expiratory pressure (MEP)
Change in maximum inspiratory pressure (MIP)
Change in motor performance
Change in peak expiratory flow (PEF)
Change in respiratory symptoms
Change in sleep quality
Change in swallowing symptoms
Change in voice quality

Trial Safety

Safety Progress

1 of 3

Other trials for Late-Onset Pompe Disease

Trial Design

0 Treatment Group

29 Total Participants · 0 Treatment Group

Primary Treatment: Treatment · No Placebo Group · N/A

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 15, week 30
Closest Location: Duke University Medical Center · Durham, NC
Photo of Duke University Medical Center 1Photo of durham 2Photo of durham 3
2003First Recorded Clinical Trial
1 TrialsResearching Late-Onset Pompe Disease
1819 CompletedClinical Trials

Who is running the clinical trial?

Genzyme, a Sanofi CompanyIndustry Sponsor
520 Previous Clinical Trials
85,151 Total Patients Enrolled
Duke UniversityLead Sponsor
2,137 Previous Clinical Trials
3,197,532 Total Patients Enrolled
Harrison Jones, PhDPrincipal InvestigatorDuke University
4 Previous Clinical Trials
118 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 6 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have access to a computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies.
You are able to understand and follow directions for study participation.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.