Clinical research studies are used to show if/how an investigational drug works and to find out if it is safe. They can also be referred to as clinical research trials. Clinical studies are run by qualified healthcare professionals. The doctors and other healthcare providers are responsible for the study-related care of the people that participate (or enroll) in these studies. Clinical research studies are a mandatory part of the process that leads to health authority review and approval which is required before a drug can be marketed.
Independent committees (called Institutional Review Boards or IRBs) are made up of medical and nonmedical people who also watch over clinical research studies to make sure that the people who enroll are properly and adequately informed and consent to study participation.
The purpose of the Baby-COMET study is to evaluate an investigational medication for the treatment of the infant form of Pompe Disease (IOPD).
If enrolled in this study, your infant will participate for up to four years with study visits approximately every two weeks. During these visits, your infant will receive the investigational medication in addition to study-related procedures, such as physical exams, blood tests, electrocardiograms, echocardiograms, and questionnaires.
The study is organized to have a screening period up to 28 days, an initial investigational treatment period lasting one year (additional 28 visits), an extension investigational treatment period lasting one year (additional 26 visits), and an extended long term investigational treatment period of two years (additional 52 visits). Following these study treatment periods your infant will have one additional study visit, approximately 4 weeks after the last dose of investigational medication, for your study doctor to make a final assessment of safety and study treatment effects. Your infant may discontinue study participation at any time.
The use of the drug in the Baby-COMET study is investigational. The safety and efficacy have not been established and are being investigated in this trial.
Your infant will have regular visits over four years with doctors and/or healthcare professionals for monitoring changes in Pompe disease and overall health. These visits are scheduled in advance and without the need for waiting for lengthy periods. Thus, there will be regular assessments and testing which ensures that any changes in their health status are identified immediately, so that they can be addressed appropriately in a timely manner.
You and your infant do not need medical insurance to participate in this study. There will be no cost for study-related exams, investigational medication or other study-related medical care. The study may offer compensation in return for travel. There may also be educational opportunities and support available throughout the study relating to Pompe disease.
Also, you should know that your infant's participation is voluntary and you will have the right to stop the participation of your infant from the study at any time. Your infant will continue to be followed-up normally, by your own doctor.
National Institute of Neurological Disorders and Stroke - Pompe Disease Information Page, 27March2019. Accessed at https://www.ninds.nih.gov/Disorders/All-Disorders/Pompe-Disease-Information-Page on 06July2021.