ClinicalTrials.Veeva

Menu

High Dose Inspiratory Muscle Training in LOPD

Duke University logo

Duke University

Status

Enrolling

Conditions

Lysosomal Disease
Late-Onset Pompe Disease

Treatments

Device: IMT therapy using the Pr02 mobile device

Study type

Interventional

Funder types

Other
Industry

Identifiers

NCT05431127
Pro00109392

Details and patient eligibility

About

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.

Full 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.

Enrollment

29 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Supportive Care

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

29 participants in 1 patient group

High Dose Inspiratory Muscle Training
Other group
Description:
Inspiratory Muscle Training 3 times a week over 26 weeks
Treatment:
Device: IMT therapy using the Pr02 mobile device

Trial contacts and locations

1

Loading...

Central trial contact

Victoria Eifert; Kelly Crisp, MA, CCC-SLP

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems