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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 15, week 30
Awards & highlights
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.
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.Check my eligibility
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.See study design
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 belowSelect...
I am 18 years old or older.
Select...
I have been diagnosed with Late-Onset Pompe Disease (LOPD).
Select...
My lung function is more than half of what's expected for my age and sex.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 15, week 30
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 15, week 30
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in maximum inspiratory pressure (MIP)
Secondary outcome measures
Change in ability to communicate
Change in compression phase duration (CPD)
Change in cough volume acceleration (CVA)
+19 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: High Dose Inspiratory Muscle TrainingExperimental Treatment1 Intervention
Inspiratory Muscle Training 3 times a week over 26 weeks
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Who is running the clinical trial?
Duke UniversityLead Sponsor
2,363 Previous Clinical Trials
3,420,457 Total Patients Enrolled
Genzyme, a Sanofi CompanyIndustry Sponsor
524 Previous Clinical Trials
85,600 Total Patients Enrolled
Harrison Jones, PhDPrincipal InvestigatorDuke University
4 Previous Clinical Trials
115 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I am 18 years old or older.I am unable to understand and give consent for medical procedures.I have had gene therapy for Pompe disease.I have been on the same Pompe disease treatment for over 6 months.I have been diagnosed with Late-Onset Pompe Disease (LOPD).I can follow study instructions.My lung function is more than half of what's expected for my age and sex.I do not have severe COPD, significant mental illness, or dementia.I use a machine to help me breathe when I'm awake.
Research Study Groups:
This trial has the following groups:- Group 1: High Dose Inspiratory Muscle Training
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does this research endeavor still require participants?
"The details of this trial, which was initially publicized on July 19th 2022 and most recently updated two days later, are presently available for recruitment purposes."
Answered by AI
How many people are currently enrolled in this experiment?
"Affirmative. According to the information presented on clinicaltrials.gov, this medical trial is presently recruiting participants and was initially posted in July 19th 2022 before being updated 2 days later. 29 patients need to be enrolled from 1 facility for the study's completion."
Answered by AI
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