High Dose Inspiratory Muscle Training in LOPD

Last updated: November 12, 2024
Sponsor: Duke University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Wolman Disease

Metachromatic Leukodystrophy - Mld

Pompe Disease

Treatment

IMT therapy using the Pr02 mobile device

Clinical Study ID

NCT05431127
Pro00109392
  • Ages > 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

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 videovisits and sensor-based respiratory technologies

Exclusion

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

Study Design

Total Participants: 29
Treatment Group(s): 1
Primary Treatment: IMT therapy using the Pr02 mobile device
Phase:
Study Start date:
July 19, 2022
Estimated Completion Date:
March 01, 2025

Study Description

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.

Connect with a study center

  • Duke University Medical Center

    Durham, North Carolina 27705
    United States

    Active - Recruiting

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