Pan African Clinical Trials Registry
South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0506 / +27 21 938 0834 Fax: +27 21 938 0836
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR201506001171421 Date of Approval: 10/06/2015
Trial Status: Registered in accordance with WHO and ICMJE standards
TRIAL DESCRIPTION
Public title The effect of cough assist and inspiratory muscle training in patients with NMD
Official scientific title The effect of mechanical insufflation-exsufflation 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.
Brief summary describing the background and objectives of the trial Patients with neuromuscular diseases have a high risk of morbidity and mortality due to acute respiratory infections and chronic respiratory compromise caused primarily by respiratory muscle weakness (Boitano 2006; Finder 2010; Panitch 2009). Inability to clear the airways effectively during coughing results in secretion retention, airway obstruction, increased work of breathing, recurrent chest infections, and ultimately respiratory failure (Bach 2003). Identifying effective, safe and cost-effective measures to optimise cough efficacy and improve inspiratory muscle strength of paediatric and adolescent patients with neuromuscular diseases is essential in order to prevent morbidity and improve overall health-related quality of life. Due to a limited evidence base for the use of mechanical insufflation-exsufflation and inspiratory muscle training in the paediatric and adolescent neuromuscular disease sub-population, randomised controlled trials are recommended. Firstly this study aims to compare the clinical effects of standard chest physiotherapy and mechanical insufflation-exsufflation to standard chest physiotherapy and manually assisted coughing in paediatric and adolescent patients (5-18 years) with neuromuscular diseases admitted to hospital for respiratory infections. The primary outcomes measures for the acute setting include duration of hospital stay, ventilatory support and supplementary oxygen. Secondly this study aims to compare, by means of a cross-over study, the clinical outcomes and health-related quality of life of a three month inspiratory muscle training programme as an adjunct to standard care (home programme) amongst paediatric and adolescent neuromuscular disease patients (5-18 years old).The primary outcomes measures for the chronic setting include respiratory infections requiring antibiotics and frequency of hospital admissions.
Type of trial RCT
Acronym (If the trial has an acronym then please provide)
Disease(s) or condition(s) being studied Musculoskeletal Diseases,Neuromuscular diseases,Paediatrics,Respiratory
Sub-Disease(s) or condition(s) being studied
Purpose of the trial Treatment: Other
Anticipated trial start date 30/11/2015
Actual trial start date
Anticipated date of last follow up 27/06/2016
Actual Last follow-up date
Anticipated target sample size (number of participants) 88
Actual target sample size (number of participants)
Recruitment status Not yet recruiting
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Parallel: different groups receive different interventions at same time during study Randomised Randomisation table created by a computer software programme Sealed opaque envelopes Masking/blinding used Outcome Assessors
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group Mechanical insufflation-exsufflation Twice a day (4 sets consisting of 5 breaths, 1-2 minutes of rest in-between sets) Whilst patient is admitted to hospital Mechanical insufflation-exsufflation (cough assist) conducted with the Nippy Clearway (pressures ranging from 10-30 cmH2O) 22
Experimental Group Inspiratory muscle training Twice a day (10 breaths, 3 sets, 20-60 seconds of rest in-between sets) Three months Inspiratory muscle training by making use of the Powerbreathe K2 handheld device 22
Control Group Standard care N/A Three months Standard management and care (including home programmes) for out-patient NMD patients. 22
Control Group Manual assisted coughing Twice a day Whilst patient is admitted to hospital Cough assist conducted with manually applying pressure to the chest. 22
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
MI-E study: Documented diagnosis of any congenital or paediatric/adolescent NMD. Patients admitted for acute respiratory infections and/or respiratory complications such as increased work of breathing, respiratory muscle fatigue, orthopnoea, retention of secretions/inability to clear secretions, decreased peak expiratory cough flow. IMT study: Documented diagnosis of any congenital or paediatric/adolescent NMD. Patients admitted for acute respiratory infections and/or respiratory complications such as increased work of breathing, respiratory muscle fatigue, orthopnoea, retention of secretions/inability to clear secretions, decreased peak expiratory cough flow. MI-E study: Unstable vital signs such as resting arterial O2 saturation (SpO2) of <90% and/or end-tidal carbon dioxide tension (PETCO2) of >7kPa on the day of recruitment. If the vital signs should stabilise within the following 48 hours, patients will be reconsidered for inclusion. If patients are terminally ill, have bullae emphysema, scoliosis > 100 degrees (La Place¿s Law) or cannot cooperate/follow basic instructions. Previous history of a pneumothorax/ pneumo-mediastinum, recent (< 6months) barotrauma, thoracic/abdominal surgery. Patient that present with cardiac failure (confirmed by a physician). Patient that are participating in the IMT study, as this might influence their reaction to MI-E treatment as well as their clinical outcomes. Patients that are unable to cooperate/comply with the cough assist technique(s). IMT study: Patients that are terminally ill. Patients with <25% predicted value VC will be excluded. Patients that are unable to cooperate/comply with the training programme. 5 Year(s) 18 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes Human Research Ethics Committee (UCT)
Ethics Committee Address
Street address City Postal code Country
Main Road, Observatory Cape Town 7937 South Africa
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome Duration of hospital stay At discharge
Primary Outcome Ventilatory support If applicable: on a daily basis
Primary Outcome Supplemental oxygen If applicable: on a daily basis
Primary Outcome Respiratory infections requiring antibiotics (frequency) End of three month IMT and at the end of the three month follow-up.
Primary Outcome Hospital admissions (frequency) End of three month IMT and at the end of the three month follow-up.
Primary Outcome Patient's functional ability At baseline and at the end of follow-up.
Secondary Outcome Disease severity scale Baseline and at discharge
Secondary Outcome Vital signs: respiratory rate, heart rate, oxygen saturation, blood gases if available (PaO2, PaCO2 and FiO2) Daily
Secondary Outcome Pulmonary function tests (VC, FVC, FEV1 and TLC) Baseline and discharge
Secondary Outcome Cough efficacy (PECF) Baseline and discharge
Secondary Outcome Comfort of breathing (VAS) Daily
Secondary Outcome Comfort of cough augmentation technique (MAC vs MI-E) (VAS) First day of treatment and last day of treatment
Secondary Outcome Vital signs: respiratory rate, heart rate, oxygen saturation, blood gases if available (PaO2, PaCO2 and FiO2) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Pulmonary function tests (VC, FVC, FEV1 and TLC) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Cough efficacy (PECF) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Inspiratory muscle strength (SNIP) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Inspiratory muscle strength (PImax) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Inspiratory muscle strength (PImax) At baseline and every two/four weeks during the follow-up visit to the hospital until the end of the trial.
Secondary Outcome Health-related quality of life (PedsQL) At baseline and at the end of the trial.
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Doctor George Mukhari Academic Hospital Molotlegi Street, Garankuwa Pretoria 0204 South Africa
Red Cross War Memorial Children's Hospital Klipfontein Road, Mowbray Cape Town 7700 South Africa
FUNDING SOURCES
Name of source Street address City Postal code Country
Research development grant (Sefako Makgatho Health Sciences University) Molotlegi Street, Garankuwa Pretoria 0204 South Africa
URC Equipment Grant (Western Cape) Main Road, Observatory Cape Town 7937 South Africa
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Anri Human Molotlegi Street, Garankuwa Pretoria 0204 South Africa University
COLLABORATORS
Name Street address City Postal code Country
Brenda Morrow Klipfontein Road, Mowbray Cape Town 7700 South Africa
Jennifer Jelsma Main Road, Observatory Cape Town 7937 South Africa
Lieselotte Corten Main Road, Observatory Cape Town 7937 South Africa
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Anri Human anrihuman@gmail.com 012 521-4047 Molotlegi Street, Garankuwa
City Postal code Country Position/Affiliation
Pretoria 0204 South Africa Lecturer
Role Name Email Phone Street address
Public Enquiries Anri Human anrihuman@gmail.com 012 521-4047 Molotlegi Street, Garankuwa
City Postal code Country Position/Affiliation
Pretoria 0204 South Africa Lecturer
Role Name Email Phone Street address
Scientific Enquiries Brenda Morrow brenda.morrow@uct.ac.za 021 658-5074 Klipfontein Road
City Postal code Country Position/Affiliation
Cape Town 7700 South Africa
REPORTING
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