High Dose Inspiratory Muscle Training in LOPD

Description

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.

Conditions

Late-Onset Pompe Disease, Lysosomal Disease

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Study Overview

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Study Details

Study overview

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.

High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)

High Dose Inspiratory Muscle Training in LOPD

Condition
Late-Onset Pompe Disease
Intervention / Treatment

-

Contacts and Locations

Durham

Duke University Medical Center, Durham, North Carolina, United States, 27705

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Duke University,

Harrison Jones, PhD, PRINCIPAL_INVESTIGATOR, Duke University

Study Record Dates

2025-03-01