High Dose Inspiratory Muscle Training in LOPD

Late-Onset Pompe Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05431127
• Other study ID #: Pro00109392
• Status: Recruiting
• Phase: N/A
• Start date: July 19, 2022
• Est. completion date: August 2024

Study information

• Verified date: July 2023
• Source: Duke University
• Contact: Kelly Crisp, MA, CCC-SLP
• Phone: 919-681-1852
• Email: kelly.crisp[@]duke.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 29
• Est. completion date: August 2024
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-Onset Pompe Disease

Intervention

Device:
• 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.

Locations

Country	Name	City	State
United States	Duke University Medical Center	Durham	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Duke University	Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in maximum inspiratory pressure (MIP)	Change pre-test to post test, measured in cm H20	Baseline, week 15, week 30
Secondary	Change in maximum expiratory pressure (MEP)	Change pre-test to post test, measured in cm H20	Baseline, week 15, week 30
Secondary	Change in inspiratory power curve (IPC)	Change pre-test to post test, measured in pressure-time units (PTUs)	Baseline, week 15, week 30
Secondary	Change in inspiratory duration (ID)	Change pre-test to post test, measured by duration in seconds	Baseline, week 15, week 30
Secondary	Change in fatigue index test score (FIT)	Change pre-test to post test, a proprietary measure which quantifies propensity to inspiratory muscle fatigue based upon the relationship between inspiratory capacity and demand using the following equation: (IPC [in Watts] x MID) / (Power500 x T500), where Power500 = power expended to inspire a mass of 500 mL air and T500 = time when mass of inspired air=500 mL at sea level	Baseline, week 15, week 30
Secondary	Change in forced vital capacity (FVC)	Change pre-test to post-test, measured in liters using a portable hand-held spirometer	Baseline, week 15, week 30
Secondary	Change in forced expiratory volume over 1 second (FEV1)	change pre-test to post-test, measured in liters per second using a portable hand-held spirometer	Baseline, week 15, week 30
Secondary	Change in peak expiratory flow (PEF)	change pre-test to post-test, measured in liters per second using portable hand-held spirometer	Baseline, week 15, week 30
Secondary	Change in inspiratory phase duration (IPD)	change pre-test to post-test, measured in seconds	Baseline, week 15, week 30
Secondary	Change in inspiratory peak flow (IPF)	Change pre-test to post-test, measured in liters per second	Baseline, week 15, week 30
Secondary	Change in compression phase duration (CPD)	Change pre-test to post-test, measured in seconds	Baseline, week 15, week 30
Secondary	Change in expiratory phase rise time (EPRT)	Change pre-test to post-test, measured in liters per second	Baseline, week 15, week 30
Secondary	Change in cough volume acceleration (CVA)	Change pre-test to post-test, measured by EPF/EPRT	Baseline, week 15, week 30
Secondary	Change in fatigue	Change pre-test to post-test, measured by Fatigue Severity Scale (FSS) survey completion, using a 7 point ordinal scale where a rating of 1 indicates strong disagreement and a rating of 7 indicates strong agreement.	Baseline, week 15, week 30
Secondary	Change in impact of fatigue on quality of life (QOL)	Change pre-test to post-test, measured by Modified Fatigue Impact Scale (MFIS) survey completion using a score of 0 (never affected) to 4 (almost always affected). The total score ranges from 0 to a maximum of 84. Higher scores indicate greater impact of fatigue on quality of life.	Baseline, week 15, week 30
Secondary	Change in daytime sleepiness	Change pre-test to post-test, measured by Epworth Sleepiness Scale (ESS) survey completion using a 0 to 3 ordinal scale in which 0=no chance of dozing, 1=slight chance of dozing, 2= moderate chance of dozing, and 3=high chance of dozing. Scores are summed to obtain total ESS score where a score >10 reflects excessive daytime sleepiness.	Baseline, week 15, week 30
Secondary	Change in sleep quality	Change pre-test to post-test, measured by Pittsburgh Sleep Quality Index (PSQI) survey completion. The PSQI is a 9-question, 19-item instrument. Items 1 to 4 are open-ended questions (customary bedtime, length of time to fall asleep). Items 5 to 8 (including the 10 questions comprising item 5) are sleep symptoms which are rated as to their frequency using an ordinal scale: 0=not occurring in the last month, 1=less than once a week, 2=once or twice a week, and 3=three or more times a week. Item 9 is a rating of overall sleep quality over the past month using a 0 to 3 ordinal scale: 0=very good; 1=fairly good; 2= fairly bad; and 3=very bad. Scores from the 19-items are combined according to standard scoring criteria to obtain a Global PSQI score. Scores >5 indicate reduced sleep quality.	Baseline, week 15, week 30
Secondary	Change in respiratory symptoms	Change pre-test to post-test, measured by Respiratory Symptoms Questionnaire (RSQ) completion using a 0-3 scale where a higher score indicates worse symptoms.	Baseline, week 15, week 30
Secondary	Change in motor performance	Change pre-test to post-test, measured by Rotterdam Handicap Scale (RHS) survey completion using a ranging scale of 1 - 4 where 1 = unable to fulfil the task or activity and 4 = complete fulfillment of the task or activity. Scores are summed and range from 9-36.	Baseline, week 15, week 30
Secondary	Change in health-related quality of life	Change pre-test to post-test, measured by Short Form 36 (SF-36) survey completion where physical and mental component summary scales (PCS and MCS) are calculated from the subscales and transformed to a normalized T-score with a mean of 50 and a standard deviation of 10. Higher scores represent better health-related quality of life.	Baseline, week 15, week 30
Secondary	Change in ability to communicate	Change pre-test to post-test, measured by Communicative Participation Item Bank-Short From (CPIB-10) survey completion using a 4-point scale (not at all=3, a little=2, quite a bit=1, very much=0). Item scores are added to obtain the summary score which ranges from 0-30. This can be transformed into a standard T score (mean=50, SD=10). Higher scores represent less interference in communication participation.	Baseline, week 15, week 30
Secondary	Change in voice quality	Change pre-test to post-test, measured by Voice Handicap Index (VHI-10) survey completion using a 5-point scale (0=never, 1=almost never, 2=sometimes, 3=almost always, 4=always). Item responses are added to obtain a total score (values >11 abnormal) with higher scores indicating greater perception of voice-related handicap.	Baseline, week 15, week 30
Secondary	Change in swallowing symptoms	Change pre-test to post-test, measured by Eating Assessment Tool (EAT-10) survey completion using a 5-point scale (0=no problem, 4=severe problem). Item responses are added to obtain a total score (values >3 abnormal) with higher scores indicating greater severity of swallowing symptoms.	Baseline, week 15, week 30