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Device

IMT Therapy for Pompe Disease

N/A
Recruiting
Led By Harrison Jones, PhD
Research Sponsored by Duke University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 18 years
Confirmed diagnosis of LOPD
Must not have
Inability to give legally effective consent
Prior history of gene therapy for LOPD
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 15, week 30
Awards & highlights
No Placebo-Only Group

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.

Who is the study for?
Adults diagnosed with Late-onset Pompe Disease (LOPD) who have been stable on their current treatment for over six months can join. They must be able to follow study instructions, have a minimum inspiratory muscle strength, and access to technology for remote participation. Those with severe lung diseases, significant mental illness, dementia or prior gene therapy for LOPD cannot participate.
What is being tested?
The trial is testing high-dose inspiratory muscle training (IMT) using the Pr02 mobile device in people with LOPD. It's done remotely and aims to check if it's safe and doable while measuring its impact on breathing function and patient-reported outcomes.
What are the potential side effects?
While specific side effects of IMT are not detailed here, potential risks may include discomfort from the use of the device or fatigue due to intense respiratory exercises.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I have been diagnosed with Late-Onset Pompe Disease (LOPD).
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My lung function is more than half of what's expected for my age and sex.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am unable to understand and give consent for medical procedures.
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I have had gene therapy for Pompe disease.
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I do not have severe COPD, significant mental illness, or dementia.
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I use a machine to help me breathe when I'm awake.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 15, week 30
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 15, week 30 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in maximum inspiratory pressure (MIP)
Secondary study objectives
Change in ability to communicate
Change in compression phase duration (CPD)
Change in cough volume acceleration (CVA)
+19 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: High Dose Inspiratory Muscle TrainingExperimental Treatment1 Intervention
Inspiratory Muscle Training 3 times a week over 26 weeks

Find a Location

Who is running the clinical trial?

Duke UniversityLead Sponsor
2,463 Previous Clinical Trials
2,981,876 Total Patients Enrolled
Genzyme, a Sanofi CompanyIndustry Sponsor
527 Previous Clinical Trials
185,721 Total Patients Enrolled
Harrison Jones, PhDPrincipal InvestigatorDuke University
4 Previous Clinical Trials
115 Total Patients Enrolled

Media Library

Pr02 mobile device (Device) Clinical Trial Eligibility Overview. Trial Name: NCT05431127 — N/A
Pompe Disease Research Study Groups: High Dose Inspiratory Muscle Training
Pompe Disease Clinical Trial 2023: Pr02 mobile device Highlights & Side Effects. Trial Name: NCT05431127 — N/A
Pr02 mobile device (Device) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05431127 — N/A
~2 spots leftby Mar 2025
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