PACTR201506001171421.

Study name	The effect of mechanical insufflation‐exsufflation (MI‐E) (and inspiratory muscle training) on clinical outcomes and health‐related quality of life in paediatric and adolescent patients with neuromuscular diseases presenting with respiratory muscle weakness: a multi‐centre trial
Methods	RCT with parallel‐group assignment
Participants	Children with NMD, aged 5–18 years Inclusion criteria Documented diagnosis of any congenital or paediatric/adolescent NMD People admitted to hospital for acute respiratory infections or respiratory complications (or both) such as increased work of breathing, respiratory muscle fatigue, orthopnoea, retention of secretions/inability to clear secretions, decreased PECF Exclusion criteria Unstable vital signs such as resting arterial SpO2 < 90% or PETCO2 > 7 kPa (or both) on the day of recruitment. If the vital signs should stabilise within the following 48 hours, patients will be reconsidered for inclusion Patients who are terminally ill Bullae emphysema Scoliosis > 100° (La Place's Law) Inability to co‐operate/follow basic instructions Previous history of a pneumothorax/ pneumo‐mediastinum Recent (< 6 months) barotrauma or thoracic/abdominal surgery Patient that present with cardiac failure (confirmed by a physician). Patient participating in the IMT study would not be eligible for the MI‐E study, as this might influence their reaction to MI‐E treatment as well as their clinical outcomes. Patients who are unable to co‐operate/comply with the cough assist technique(s)
Interventions	Standard management with bi‐daily MI‐E (using Nippy Clearway CoughAssist device) during hospital admission (Pi/Pe 10–30 cmH2O; 4 sets consisting of 5 breaths, 1–2 min of rest between sets Standard management with bi‐daily MAC (thoracic compressions) during hospital admission
Outcomes	Primary outcomes Duration of hospital stay Requirement for ventilatory support Oxygen requirement Secondary outcomes Disease severity scale Vital signs: respiratory rate, heart rate, oxygen saturation, blood gases if available (PaO2, PaCO2 and FiO2) Pulmonary function tests (baseline to discharge) Cough efficacy (PECF) (measured at baseline and discharge) Comfort of breathing (VAS) (measured daily) Comfort of cough augmentation technique (MAC vs MI‐E) (VAS) (measured at first and last intervention)
Starting date	July 2016
Contact information	Anri Human; anrihuman@gmail.com Brenda Morrow (PhD); brenda.morrow@uct.ac.za
Notes	Only the MI‐E arm (and not the IMT intervention) of this study would potentially be eligible for inclusion in updates of this review. The PACT site states that the study has not started recruiting; however the study has enrolled participants but is temporarily halted owing to feasibility issues. Data are not yet available for analysis.

AUC: area under the curve; bpm: beats per minute; FiO₂: fraction of inspired oxygen; IMT: inspiratory muscle training; MAC: manually assisted cough; MI‐E: mechanical insufflation‐exsufflation; min: minute; NMD: neuromuscular disease; PaCO₂: partial pressure of carbon dioxide; PACT: Pan African Clinical Trials; PCF: peak cough flow; Pe: exsufflation pressure; PECF: peak expiratory cough flow; PETCO₂: end‐tidal carbon dioxide tension; Pi: insufflation pressure; RCT: randomised controlled trial; SpO₂: peripheral capillary oxygen saturation; Te: time for exsufflation; Ti: time for insufflation; VAS: visual analogue scale.