NCT01701154

Brief Summary

The purpose of the study is to determine if respiratory muscle strength training will be beneficial for inspiratory and expiratory muscle strength in adults and children with Pompe disease.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
11

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Dec 2010

Typical duration for all trials

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

Study Start

First participant enrolled

December 1, 2010

Completed
1.8 years until next milestone

First Submitted

Initial submission to the registry

October 3, 2012

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 4, 2012

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 15, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 15, 2012

Completed
Last Updated

May 30, 2023

Status Verified

May 1, 2023

Enrollment Period

2 years

First QC Date

October 3, 2012

Last Update Submit

May 25, 2023

Conditions

Pompe Disease

Keywords

Pompe respiratory

Outcome Measures

Primary Outcomes (1)

  • Change in maximum inspiratory pressure

    change from pre-test to post-test

    baseline, week 12

Study Arms (1)

Pompe

Adults and children with Pompe disease.

Other: respiratory muscle strength training

Interventions

respiratory muscle strength trainingOTHER
Pompe

Eligibility Criteria

Age3 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adults and children with Pompe disease

You may qualify if:

  • include skin fibroblast acid α-glucosidase (GAA) activity ≤1% of the normal mean, a minimum age of 3 years, the ability to participate in an intensive RMST research program, and the ability to maintain a consistent amount of nonresearch related physical activity over the course of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Health System

Durham, North Carolina, 27710, United States

Location

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

    Duke University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 3, 2012

First Posted

October 4, 2012

Study Start

December 1, 2010

Primary Completion

December 15, 2012

Study Completion

December 15, 2012

Last Updated

May 30, 2023

Record last verified: 2023-05

Locations

US(1)