Spinal Muscular Atrophy Randomized Controlled Trial on Cough Techniques (Patient tailored airway clearance in patients with Spinal Muscular Atrophy: a randomized controlled trial comparing air stacking and mechanical insufflation-exsufflation.)

Conditions: Spinal muscular atrophy (SMA)
10028396
10024970

Registration Number: NL-OMON56529

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 46

Inclusion Criteria

Patients with Spinal Muscular Atrophy
Peak expiratory cough flow < 270 L/min if >= 10 years or PECF <200 L/min in
patients 8-9 years
Patients from 8 years of age.
Able to perform spirometry

Exclusion Criteria

- Severe gastroesophageal reflux with risk of aspiration despite treatment
- Severe esophageal and gastric varices
- Recent pneumothorax (< 6 weeks)
- Recent barotrauma
- Emphysema, bullae
- Tracheo-oesphageal fistula
- Severe facial deformity
- Tracheostomy
- Patient or legal representative unable to speak and understand Dutch or
English
- RTI in 6 weeks prior to inclusion. If the patient suffers from a RTI in the
period between screening and baseline measurements, it is not required to
repeat the screening.
- Respiratory muscle training initiated < 6 weeks prior to inclusion
-Daily use of mechanical insufflation-exsufflation.Patients who used MI-E
temporarily during a respiratory tract infection in the past are eligible for
inclusion.*

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary endpoint is the immediate effect on improvement of Peak Expiratory<br /><br>Cough Flow (PECF) over a period of 3 years</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints are the number of respiratory tract infections (RTIs)<br /><br>requiring hospital admissions or antibiotics, patient reported patient<br /><br>satisfaction, lung function decline, patient satisfaction, adverse events and<br /><br>compliance to treatment</p><br>