NCT02801539

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
22

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Feb 2017

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 13, 2016

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 16, 2016

Completed
8 months until next milestone

Study Start

First participant enrolled

February 2, 2017

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 22, 2018

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 17, 2019

Completed
Last Updated

July 30, 2019

Status Verified

July 1, 2019

Enrollment Period

1.7 years

First QC Date

June 13, 2016

Last Update Submit

July 29, 2019

Conditions

Glycogen Storage Disease Type II

Outcome Measures

Primary Outcomes (2)

  • 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).

    baseline, 12 weeks, 6 months, 9 months

  • 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.

    12 weeks

Secondary Outcomes (6)

  • Change in maximum expiratory pressures (MEP)

    baseline, 12 weeks, 6 months, 9 months

  • Change in 6-minute walk test (6MWT)

    baseline, 12 weeks, 6 months, 9 months

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

    baseline, 12 weeks, 6 months, 9 months

  • Change in peak cough flow (PCF)

    baseline, 12 weeks, 6 months, 9 months

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

    baseline, 12 weeks, 6 months, 9 months

  • +1 more secondary outcomes

Study Arms (2)

Respiratory muscle training (RMT)

EXPERIMENTAL

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

Device: RMT therapy using modified RMT device

Sham-RMT

SHAM COMPARATOR

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

Device: Sham-RMT therapy using modified RMT device

Interventions

RMT therapy using modified RMT deviceDEVICE

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

Respiratory muscle training (RMT)
Sham-RMT therapy using modified RMT deviceDEVICE

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

Sham-RMT

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Related Publications (1)

  • 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.

    PMID: 31303277DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Harrison Jones, PhD

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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
SUPPORTIVE CARE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 13, 2016

First Posted

June 16, 2016

Study Start

February 2, 2017

Primary Completion

October 22, 2018

Study Completion

April 17, 2019

Last Updated

July 30, 2019

Record last verified: 2019-07

Data Sharing

IPD Sharing
Will not share

Locations

US(1)