University of British Columbia SUBJECT INFORMATION AND CONSENT FORM TITLE OF STUDY: A comparison of three methods for improving expiratory cough flow and lung volume in children with neuromuscular diseases PRINCIPAL INVESTIGATOR: Mike Seear, MD Clinical Professor Divisional Head of Critical Care Department of Pediatrics University of British Columbia British Columbia Children s Hospital Phone: 604 875 2000 Co Investigators: Maggie McIlwaine, physiotherapist Hilda Perry, physiotherapist Melissa Richmond, MD Kathryn Selby, MD Connie Yang, MD INSTITUTION: British Columbia s Children s Hospital CONTACT NUMBERS: EMERGENCY CONTACT: For any emergency related to your child s neuromuscular disease please contact the paging service at 604 875 2161 and ask to have the neurologist on call paged. This service is available 24 hours a day, 7 days a week. If you have any questions regarding this study, you may contact Dr Seear at 604 875 2119. If you have any concerns about your rights as a research subject and/or your experiences while participating in this study, contact the Research Complaint line in the University of British Columbia Office of Research Services by email at RSIL@ors.ubc.ca or by phone at 604 822 8598 or toll free at 1 877 822 8598. Version 1 22 February 2013 Page 1 of 6
If you are a parent or legal guardian of a child who may take part in this study, permission from you is required. When we say you or your in this consent form, we mean you and/or your child; we means the doctors and other staff. Introduction You are being invited to take part in this research study because you have a neuromuscular disease and have weak chest muscles which make it difficult for you to clear your lungs, which make you more likely to get a chest infection. This study is looking at three different treatments to see which treatment is most effective in helping to clear the lungs in a short term. Background Children with neuromuscular disease often have weak muscles and low muscle tone. The primary effects of weakness are: poor thoracic muscle and diaphragm function, low tone in the muscles of swallowing and also in those muscles providing support for the spine. The results of this weakness are: hypoventilation (slow shallow breathing), steady loss of lung volume, recurrent chest infections due to ineffective cough and secretion clearance, frequent development of progressive scoliosis (curvature of the spine) and poor airway protection with recurrent aspiration (inhaling into the lungs) of oral secretions or food. The final result can be respiratory failure. Although there are many devices and treatment that have been proposed for improving secretion clearance and lung function in very weak patients, there has been little research done to determine whether any one of these techniques has any measurable benefit. Purpose of the Research This is a pilot study. A pilot study is a smaller version of a large study that is done prior to undertaking a larger study. In this study we have identified three treatments that we would like to compare, to see which one is most effective in the short term in helping to improve lung volume and clearing secretions out of the lung. Your Participation is Voluntary Your participation is entirely voluntary, so it is up to you to decide whether or not to take part in this study. Before you decide, it is important for you to understand what the research involves. This consent form will tell you about the study, why the research is being done, what will happen to your child during the study and the possible benefits, risks and discomforts. If you wish to take part in this study, you will be asked to sign this form. If you decide you will take part in this study, you are still free to withdraw at any time, without giving any reasons for your decision. Version 1 22 February 2013 Page 2 of 6
If you do not wish to take part, you do not have to provide any reason for your decision not to participate nor will you lose the benefit of any medical care to which you are entitled or are presently receiving. Please take time to read the following information carefully and consider discussing it with your family, friends, and doctor before you decide. Who is conducting the study? This study is being carried out at British Columbia s Children s Hospital (BCCH) by Dr. Mike Seear, the Principal Investigator. The study is not receiving funds from an external agency or sponsor Who Can Participate in the Research? Children diagnosed with a neuromuscular disease, who are able to use a face mask and can cognitively perform lung function testing (PFTs pulmonary lung function). Who cannot Participate in the Research? You cannot participate if you: have a tracheostomy are unable to have a good face mask seal are unable to perform a lung function test What does this study involve? After you have signed this consent and agree to take part in this study you will have your weight and height measured, if this is unable to be done due to severe spinal curvature, arm span will be used instead. The study involves receiving three different types of treatment to help lung function over three consecutive days. Prior to each treatment you will be given a standard lung function test. Testing will be organized so that the process takes three consecutive mornings, i.e. 3 to 4 hours a day, for 3 days. All three methods of treatment will be administered by a physiotherapist experienced in the use of these machines. Each one is a commonly used device that has been used in various centres for many years. The techniques are summarized below but they have several features in common. Each one provides an active pressure assisted inspiratory 9 in breath) breath followed by an assisted expiration (out breath) and cough. All also deliver the assisted breath using a face mask. Bipap assisted breath stacking (BABS) After receiving instructions, you will be asked to take a deep breath through a face mask and allow a machine called bipap to help you increase the volume of air you take in. You will then need to hold your breath, and then release it. You will be treated with three cycles of three breaths. After each cycle you will pause and perform huffing and assisted coughing. The physiotherapist will help you do Version 1 22 February 2013 Page 3 of 6
this and may also need to use a suction tube to help remove any lung secretions that you coughed up. Mechanical insufflations exsufflation (MI E) After receiving instructions, you will be asked to breathe through a mask and allow a machine called the CoughAssist In Exsufflator to assist your breathing. The machine will gradually inflate your lungs (called insufflations). Once the lungs are full, it will assist you to breathe out using a slight suction pressure through the face mask. This assisted exhalation will cause you to cough and thus help to remove secretions from your lung. After each cycle you will pause and perform, huffing and assisted coughing. The physiotherapist will help you do this and may also need to use a suction tube to help remove any lung secretions coughed up. Combined oscillation and nebulisation (CON) After receiving instructions, you will be asked to breathe through a face mask attached to a machine called an oscillating PEP device. This machine creates pulses of air pressure through the mask. As you breathe against this pressure, the vibrations will help to loosen mucous and secretions in your lungs. During each treatment cycle you will also receive misted saline through the machine. After each cycle you will pause and perform, huffing and assisted coughing. The physiotherapist will help you do this and may also need to use a suction tube to help remove any lung secretions coughed up. At the end of each treatment you will receive a standard pulmonary function test to assess how your lungs are doing at 30 minutes, one and two hours after the treatment. The following information will be collected from your medical record: Age and gender Diagnosis Weight and height Risks and Discomforts All three techniques involve breathing against a positive pressure through a face mask. This is an unfamiliar sensation so a few children take time to become accustomed to the tight fitting mask and extra pressure during breathing. We start by explaining the technique carefully and build up the pressure slowly. Almost all children are able to tolerate these procedures without significant discomfort. Many children with NMDs, use these devices 3 or 4 times a day without problems. The only potential significant side effect is a leak of air from the lungs caused by the pressure. This is called a pneumothorax. While it is a theoretical problem, there have been no reports of a pneumothorax associated with the use of any of these devices. To minimize the risk, we use maximum pressures that are below the manufacturers recommendations. Version 1 22 February 2013 Page 4 of 6
Signing this consent form in no way limits you or your child s legal rights against the investigators, or anyone else, and you do not release the study doctors or participating institutions from their legal and professional responsibilities. Potential Benefits You may not receive any benefit from taking part in this study. We hope that the information learned from this study will help to establish better ways of treating respiratory problems in children with NMD in the future.. What are the alternatives to the study treatment? If you choose not to participate in this study or withdraw at a later date, you will continue to receive treatment for your NMD respiratory issues. You can discuss this with your doctor before deciding whether or not to participate in this research project. What will the Study Cost me? There is no cost to you to participate in this study. Please note you will not be paid if you choose to take part in this study. We will cover the cost of hospital parking for 3 days. What happens if I decide to withdraw my consent to participate? Your participation in this research is entirely voluntary. You may withdraw from this study at any time, and without giving any reasons for your decision. If you decide to enter the study and withdraw any time in the future, there will be no penalty or loss of benefits to you to which you are otherwise entitled, and your future medical care will not be affected. What if new information becomes available that may affect my decision to participate? If you choose to enter into this study and at a later date a more effective treatment becomes available, it will be discussed with you. You will also be advised of any new information that becomes available that may affect your willingness to remain in the study. Confidentiality Your confidentiality will be respected. However, research records and medical records identifying you may be inspected in the presence of the Investigator or his or her designate by representatives of Health Canada, and University of British Columbia Research Ethics Boards. No information or records that disclose your identity will be published without your consent, nor will any information or records that disclose your identity be removed or released without your consent unless required by law. You will be assigned a unique study number as a subject in this study. Only this number will be used on any research related information collected about you during the course of this study, so that your identity [i.e. your name or any other information that could Version 1 22 February 2013 Page 5 of 6
identify you] as a subject in this study will be kept confidential. Information that contains your identity will remain only with the Principal Investigator and/or designate. The list that matches your name to the unique identifier that is used on your researchrelated information will not be removed or released without your consent unless required by law. Your and your child s rights to privacy are legally protected by federal and provincial laws that require safeguards to insure that your privacy is respected and also give you and your child the right of access to the information about you and your child that has been provided to the investigator, and if need be to correct any errors in this information. Further details about these laws are available on request to your study doctor. Version 1 22 February 2013 Page 6 of 6