Clinical trial NCT05431127: High Dose Inspiratory Muscle Training in LOPD
Official title: High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Late-Onset Pompe Disease
Lysosomal Disease
Keywords: LOPD
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: Genzyme, a Sanofi Company
First posted: 2022-06-24
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in maximum inspiratory pressure (MIP)
timeFrame: Baseline, week 15, week 30
Secondary outcomes:
measure: Change in maximum expiratory pressure (MEP)
timeFrame: Baseline, week 15, week 30
measure: Change in inspiratory power curve (IPC)
timeFrame: Baseline, week 15, week 30
measure: Change in inspiratory duration (ID)
timeFrame: Baseline, week 15, week 30
measure: Change in fatigue index test score (FIT)
timeFrame: Baseline, week 15, week 30
measure: Change in forced vital capacity (FVC)
timeFrame: Baseline, week 15, week 30
measure: Change in forced expiratory volume over 1 second (FEV1)
timeFrame: Baseline, week 15, week 30
measure: Change in peak expiratory flow (PEF)
timeFrame: Baseline, week 15, week 30
measure: Change in inspiratory phase duration (IPD)
timeFrame: Baseline, week 15, week 30
measure: Change in inspiratory peak flow (IPF)
timeFrame: Baseline, week 15, week 30
measure: Change in compression phase duration (CPD)
timeFrame: Baseline, week 15, week 30
measure: Change in expiratory phase rise time (EPRT)
timeFrame: Baseline, week 15, week 30
measure: Change in cough volume acceleration (CVA)
timeFrame: Baseline, week 15, week 30
measure: Change in fatigue
timeFrame: Baseline, week 15, week 30
measure: Change in impact of fatigue on quality of life (QOL)
timeFrame: Baseline, week 15, week 30
measure: Change in daytime sleepiness
timeFrame: Baseline, week 15, week 30
measure: Change in sleep quality
timeFrame: Baseline, week 15, week 30
measure: Change in respiratory symptoms
timeFrame: Baseline, week 15, week 30
measure: Change in motor performance
timeFrame: Baseline, week 15, week 30
measure: Change in health-related quality of life
timeFrame: Baseline, week 15, week 30
measure: Change in ability to communicate
timeFrame: Baseline, week 15, week 30
measure: Change in voice quality
timeFrame: Baseline, week 15, week 30
measure: Change in swallowing symptoms
timeFrame: Baseline, week 15, week 30
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Harrison Jones, PhD
role: PRINCIPAL_INVESTIGATOR
References: