High Dose Inspiratory Muscle Training in LOPD

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-19

Study Completion Date

2025-01-01

Brief Summary

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Study Objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and 2) determine its effects on respiratory and patient-reported outcomes.

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Detailed Description

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This study aims to develop treatments that enhance respiratory strength and function to provide meaningful clinical improvements for people with LOPD. Identification of a cost-effective adjunctive intervention to address respiratory weakness remains critical to reduce disease burden, ease activity limitations and participation restrictions, and improve health-related quality of life. The proposed study will provide a high-dose inspiratory muscle training (IMT) stimulus to enhance treatment efficacy and efficiency. Our hypothesis is that high-dose IMT is necessary to produce meaningful changes in respiratory muscle strength and other outcomes in participants with LOPD.

Conditions

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Late-Onset Pompe Disease Lysosomal Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

NONE

Study Groups

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High Dose Inspiratory Muscle Training

Inspiratory Muscle Training 3 times a week over 26 weeks

Group Type OTHER

IMT therapy using the Pr02 mobile device

Intervention Type DEVICE

Inspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises.

Interventions

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IMT therapy using the Pr02 mobile device

Inspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises.

Intervention Type DEVICE

Eligibility Criteria

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Inclusion Criteria

* Age ≥ 18 years
* Confirmed diagnosis of LOPD
* MIP \>50% of predicted for sex and age
* Stable on current Pompe disease treatment regimen \>6 months
* Able to follow directions for study participation
* Access to computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies

Exclusion Criteria

* Presence of medical comorbidities that prevent meaningful study participation (e.g., COPD GOLD III-IV, significant mental illness, dementia)
* Use of continuous invasive or non-invasive ventilation while awake
* Prior history of gene therapy for LOPD
* Inability to give legally effective consent
* Inability to read and understand English

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No