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Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

Primary Purpose

Glycogen Storage Disease Type II

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

RMT therapy using modified RMT device

Sham-RMT therapy using modified RMT device

About this trial

This is an interventional supportive care trial for Glycogen Storage Disease Type II

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age ≥ 18 years
Diagnosis of LOPD
On enzyme replacement therapy for ≥ 26 weeks at pretest
Able to follow directions for study participation
Able to complete a home-based RMT regimen

Exclusion Criteria:

Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator
Inability to give legally effective consent
Inability to read and understand English

Sites / Locations

Duke University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

Respiratory muscle training (RMT)

Sham-RMT

Arm Description

Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.

Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.

Outcomes

Primary Outcome Measures

Change in respiratory strength

RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).

Feasibility of sham-RMT, as measured by program adherence

As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.

Secondary Outcome Measures

Change in maximum expiratory pressures (MEP)

Change pretest to posttest

Change in 6-minute walk test (6MWT)

Change pretest to posttest

Change in Gait, Stairs, Gower, and Chair (GSGC) scale

Change pretest to posttest

Change in peak cough flow (PCF)

Change pretest to posttest

Change in Rasch-built Pompe-specific Activity Score (R-PAct)

Change pretest to posttest

Change in diaphragm thickness

change pretest to posttest

Full Information

NCT ID

NCT02801539

First Posted

June 13, 2016

Last Updated

July 29, 2019

Sponsor

Duke University

Collaborators

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

1. Study Identification

Unique Protocol Identification Number

NCT02801539

Brief Title

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

Official Title

Respiratory Muscle Training in Late-Onset Pompe Disease

Study Type

Interventional

2. Study Status

Record Verification Date

July 2019

Overall Recruitment Status

Completed

Study Start Date

February 2, 2017 (Actual)

Primary Completion Date

October 22, 2018 (Actual)

Study Completion Date

April 17, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Duke University

Collaborators

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Glycogen Storage Disease Type II

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

22 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Respiratory muscle training (RMT)

Arm Type

Experimental

Arm Description

Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.

Arm Title

Sham-RMT

Arm Type

Sham Comparator

Arm Description

Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.

Intervention Type

Device

Intervention Name(s)

RMT therapy using modified RMT device

Intervention Description

The exercises provided by the RMT device is intended to strengthen breathing muscles.

Intervention Type

Device

Intervention Name(s)

Sham-RMT therapy using modified RMT device

Intervention Description

The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.

Primary Outcome Measure Information:

Title

Change in respiratory strength

Description

RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Feasibility of sham-RMT, as measured by program adherence

Description

Time Frame

12 weeks

Secondary Outcome Measure Information:

Title

Change in maximum expiratory pressures (MEP)

Description

Change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Change in 6-minute walk test (6MWT)

Description

Change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Change in Gait, Stairs, Gower, and Chair (GSGC) scale

Description

Change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Change in peak cough flow (PCF)

Description

Change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Change in Rasch-built Pompe-specific Activity Score (R-PAct)

Description

Change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

Title

Change in diaphragm thickness

Description

change pretest to posttest

Time Frame

baseline, 12 weeks, 6 months, 9 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age ≥ 18 years Diagnosis of LOPD On enzyme replacement therapy for ≥ 26 weeks at pretest Able to follow directions for study participation Able to complete a home-based RMT regimen Exclusion Criteria: Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator Inability to give legally effective consent Inability to read and understand English

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Harrison Jones, PhD

Organizational Affiliation

Division of Head and Neck Surgery & Communication Sciences, Duke University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

31303277

Citation

Jones HN, Kuchibhatla M, Crisp KD, Hobson Webb LD, Case L, Batten MT, Marcus JA, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial. Mol Genet Metab. 2019 Aug;127(4):346-354. doi: 10.1016/j.ymgme.2019.05.001. Epub 2019 May 8.

Results Reference

derived

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Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

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