The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…
ID
Source
Brief title
Condition
- Musculoskeletal and connective tissue disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary outcome parameter is the change in maximal inspiratory pressure
(MIP) after active inspiratory muscle training.
Secondary outcome
Several secondary outcome parameters:
* Diaphragm ultrasound (thickness, thickening, excursion).
* Pulmonary function tests (peak cough flow, forced vital capacity, forced
expiratory volume the first second, peak expiratory flow, vital capacity)
* Respiratory muscle function tests (sniff nasal inspiratory pressure, maximal
expiratory pressure, twitch mouth pressure)
* Neurological examination
* Maximal voluntary contraction
* Rate of muscle relaxation
* Mobility: Rivermead mobility index, and Community Balance and Mobility Scale
* Falls: Falls efficacy scale and Falls History Questionnaire
Background summary
Nemaline myopathy is a group of congenital, hereditary neuromuscular disorders
with variable symptoms such as muscle weakness, swallowing dysfunction, and
dysarthria. Respiratory failure is the main cause of death in nemaline myopathy
and occurs even in ambulant patients who otherwise appear to be only mildly
affected; respiratory muscle weakness may even be the presenting feature.
Inspiratory muscle training has shown to increase inspiratory muscle strength
in patients with other neuromuscular disorders. We hypothesize that inspiratory
muscle training improves inspiratory muscle function in nemaline myopathy
patients with respiratory muscle weakness.
Study objective
The primary objective is to determine the effect of a 8-week inspiratory muscle
training program on respiratory muscle function in nemaline myopathy patients.
The secondary objective is to determine respiratory muscle function in nemaline
myopathy patients and its correlation with clinical severity and general
neuromuscular function.
Study design
The study consist of two phases. Phase 1: A screening phase with an open design
from which patients will be selected for the second phase. Phase 2: A
controlled before-after trial of inspiratory muscle training. The 2 conditions
tested are active IMT and sham IMT.
Intervention
Active IMT consists of 15 minutes of IMT, twice a day, 5 days per week for 8
weeks, at a training workload of 30% of MIP using a resistive inspiratory
muscle training device. Sham IMT consists of a similar training regime using a
resistive inspiratory muscle training device where the resistance has been
removed.
Study burden and risks
The intervention of inspiratory muscle training is not associated with any
risks, but can be challenging in patients with respiratory muscle weakness to
perform. There will be four visits to the hospital in 16 weeks. The first visit
will be approximately 3.5 hours (including breaks) and the other three visits
1.5 hour. During these visits several tests and physical examinations will be
performed. Some of the tests may cause some physical discomfort, but none of
them carry any risk. Patients may benefit from participating in this study by
developing improved respiratory muscle function as a result of the inspiratory
muscle training.
Reinier Postlaan 4
Nijmegen 6525GC
NL
Reinier Postlaan 4
Nijmegen 6525GC
NL
Listed location countries
Age
Inclusion criteria
* genetically-confirmed nemaline myopathy
* informed consent from participant or legal representative
Exclusion criteria
* history of another condition that affects respiratory muscle strength or function (e.g. COPD)*
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL65214.091.18 |