E74.0
Conditions
Interventions
Group 1: All patients will perform an inspiratory muscle training on five days a week. The trainings will be performed using an electronic device that gives a feedback about the success of the trainin
Sponsors
Friedrich-Baur-Insitut
Eligibility
Sex/Gender
All
Age
18 Years to 85 Years
Inclusion criteria
Inclusion criteria: 1. The patient is willing and able to provide signed informed consent. 2. The patient has acid a-glucosidase [GAA] enzyme deficiency confirmed by GAA gene mutations. 3. The patient is able and willing to perform pulmonary lung function tests (PFT) throughout the study as well as blood draws for capillary blood gas analysis (pO2, pCO2), fill in a diary and answer questionnaires.
Exclusion criteria
Exclusion criteria: A patient who meets any of the following criteria will be excluded from this study. 1. The patient requires invasive-ventilation (non-invasive ventilation is allowed). 4. The patient is participating in another clinical study using investigational treatment. 5. The patient cannot perform pulmonary lung function tests (PFT). 6. The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of this study is to determine the safety of repetitive respiratory muscle training on respiratory muscle strength as measured by side effects and MIP (maximum inspiratory pressure) in the upright position. Side effects will be collected during the whole study, effects on MIP will be measured by change from baseline to last visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| The Secondary objectives of this study are • to evaluate the effect on FVC, FEV1, MEP and capillary blood gas analysis within a six-week-training period • to evaluate the effect on FVC, FEV1, MEP and capillary blood gas analysis within a six-week-detraining period • to evaluate the effect within a six-week- respiratory muscle training period on functional endurance (6MWT) and in quality of life, measured by health-related quality of life questionnaires (SF-36), the St. George's Respiratory Questionnaire (SGRQ) and the Modified Medical Research Council (MMRC) for Dyspnea-Score • to evaluate the effect within a six-week-detraining period on functional endurance (6MWT) and in quality of life, measured by health-related quality of life questionnaires (SF-36), the St. George's Respiratory Questionnaire (SGRQ) and the Modified Medical Research Council (MMRC) for Dyspnea-Score. | — |
Countries
Germany
Contacts
Public ContactStephan Wenninger
Friedrich-Baur-Insitut
Outcome results
None listed