The aim of this study is to assess the feasibility and efficacy of respiratory muscle training (RMT) in patients with SMA and respiratory muscle weakness.
ID
Source
Brief title
Condition
- Neurological disorders congenital
- Congenital respiratory tract disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
We hypothesize that an individualized incremental home based respiratory muscle
training program will be feasible (good adherence and good acceptability) and
will improve inspiratory and expiratory muscle strength (primary outcome
measure) and patient reported breathing difficulties, the onset of respiratory
infections and health related quality of life (secondary outcome measure) in
patients with SMA.
Secondary outcome
Reporterd airway problems by patients
Number of airway infections
Health related quality of life.
Evaluation experience trainingsperiod.
Background summary
Spinal Muscular atrophy (SMA) is characterised by progressive and predominantly
proximal and axial muscle atrophy and weakness. Weakness of the respiratory
muscles results in nocturnal hypoventilation, weak cough and ultimately
respiratory failure in the most severely affected patients, which can lead to
premature death. Treatment strategies that slow down the decline or improve
respiratory muscle function are therefore needed.
Study objective
The aim of this study is to assess the feasibility and efficacy of respiratory
muscle training (RMT) in patients with SMA and respiratory muscle weakness.
Study design
The effect of RMT will be investigated with a single blinded randomized
sham-controlled cross over trial consisting of a 4 months training period
followed by 4 months of sham-controlled training
Study burden and risks
The risks of the tests and training are negligible. Similar tests and training
methods used in usual care are well tolerated in other patients groups with
neuromuscular disorders. *
*
Heidelberglaan 100
Utrecht 3584CX
NL
Heidelberglaan 100
Utrecht 3584CX
NL
Listed location countries
Age
Inclusion criteria
* Patients with SMA aged >= 8 years with respiratory muscle weakness (Maximal
inspiratory pressure < -80 cmH2O) will be invited to participate.
Both patients without respiratory support and patients on night-time
non-invasive ventilatory support are eligible.
* Patients who are treated with Spinraza® for more than 2 months and patients
who are not/or will not be treated with Spinraza® .
Exclusion criteria
* tracheostomy,
* inability to perform respiratory and/or lung-function testing,
* inability to understand Dutch or English,
* patients who are mentally incompetent,
* patients with a history of pneumothorax or symptomatic low cardiac output
syndrome
* patients who start Spinraza during trial
Design
Recruitment
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 | NL73280.041.20 |