Clinical Trials Logo
  1. Home
  2. Pompe Disease
  3. NCT02354664

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease — DRMF  

Pompe Disease Clinical Trial

Official title:
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Clinical Trial Summary

This study intends to evaluate dynamic respiratory motor performance as a valuable measure of pulmonary function in adults with late-onset Pompe disease. The investigators will adopt a strategy that includes comprehensive evaluations of respiratory volume, flow, and timing parameters during resting and loaded breathing. These evaluations will then be associated to the standard clinical measure of maximal inspiratory pressure, the static inspiratory muscle function, as well as magnetic resonance imaging of thoracic expansion and diaphragmatic descent at rest and with exertion. Outcomes in participants with late-onset Pompe disease will be contrasted to the function of age- and gender-matched control subjects. This approach will enable the investigators to evaluate the relationship between dynamic diaphragmatic function and respiratory motor function.

Clinical Trial Description

The following tests will be completed over a two-day period: Respiratory pressure tests, breathing test, magnetic resonance imaging and magnetic resonance spectroscopy (MRI and MRS). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02354664
Study type Observational
Source University of Florida
Contact
Status Completed
Phase
Start date April 2015
Completion date October 19, 2016
View Details
See also
  Status Clinical Trial Phase
Completed NCT01942590 - Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy Phase 1/Phase 2
Not yet recruiting NCT01409486 - Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry N/A
Completed NCT00976352 - Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease Phase 1/Phase 2
Recruiting NCT01665326 - Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Completed NCT01758354 - Newborn Screening Assay of Pompe's Disease N/A
Recruiting NCT00231400 - Pompe Disease Registry Protocol
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT04476550 - Clinical Specimen Collection From Pompe Disease Patients
Completed NCT01380743 - Drug-drug Interaction Study Phase 2
Recruiting NCT05734521 - Avalglucosidase Alfa Pregnancy Study
Completed NCT02742298 - Pompe Disease QMUS and EIM N/A
Terminated NCT02185651 - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction Phase 1
Completed NCT02654886 - Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease. N/A
Completed NCT02405598 - Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease Phase 4
Completed NCT00701129 - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease Phase 4
Completed NCT01451879 - Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies N/A
Completed NCT02240407 - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease Phase 1
Completed NCT05073783 - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A