Viacyte Trial - Patient Information SheetTitle: A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study with Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01 Combination Product in Subjects with Type 1 Diabetes Mellitus Short Title: VC01-101 Principal Investigator: Dr. James Shapiro Phone number: 780-407-7330 Co-Investigator: Dr. Peter Senior Phone number: 780-407-1480 Dear Patient, I am writing to tell you about a trial being conducted by the University of Alberta at the University of Alberta Hospital (UAH) using stem cells implanted using a porous delivery capsule. The purpose of this research study is to assess the safety, tolerability, and effectiveness of a new delivery system as well as the stem cells. You may be eligible for this study if you are an adult (between the ages of 18 and 55 years old), have been diagnosed with type 1 diabetes mellitus for a minimum of 3 years, and have a stable, optimized diabetic regimen for at least 3 months. If you are interested in learning more about this study, please review the enclosed information, and complete the enclosed form. You can (or have your diabetes physician) fax this letter to 780-407-6933. You can also call us at 780-407-7330 or 780-407-1480. You can also email Dr. Andrew Malcolm (Operations Director Clinical Trials) amalcolm@ualberta.ca for more information. It is important to know that this letter is not to tell you to join this study. It is your decision. Your participation is voluntary. Whether or not you participate in this study will have no effect on your relationship with your diabetes doctor as a patient. You do not have to respond if you are not interested in this study. Thank you for your time and consideration. Sincerely, Enclosures: Opt-In Form Information Sheet Letter of Interest Date: 29 April 2015 Page 2 of 5 Opt-In Form Title: A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study with Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01 Combination Product in Subjects with Type 1 Diabetes Mellitus Short Title: VC01-101 Principal Investigator: Dr. James Shapiro Phone number: 780-407-7330 Co-Investigator: Dr. Peter Senior Phone number: 780-407-1480 Please complete this form and fax to 780-407-6933. I am interested in learning more about this study. Please contact me using the following information: Name: __________________________________________________________ Telephone(s): _____________________________________________________ Best time and day to call:____________________________________________ Email: _____________________________________@____________________ Letter of Interest Date: 29 April 2015 Page 3 of 5 Patient Information Sheet WHY IS THIS STUDY BEING CONDUCTED? As you know, management of type 1 diabetes involves constant monitoring of your blood glucose levels, typically followed by insulin injections. This process can be painful, inconvenient, and, for some, an inadequate way to treat their diabetes. In this study, doctors want to evaluate the safety, tolerability, and effectiveness of the investigational VC-01 combination product, which is implanted below the skin in the lower back or sides of the lower body in an outpatient procedure. The results from this study will provide doctors and researchers with more information about VC-01 and whether it could one day provide freedom from type 1 diabetes. NOTES ABOUT THE INVESTIGATIONAL COMBINATION PRODUCT VC-01 is called a combination product because it is made up of pancreatic endoderm cells derived from human embryonic stem cells (PEC-01) that are stored in a customized drug delivery system known as Encaptra. After VC-01 is implanted, the PEC-01 cells are expected to mature over several months and then produce insulin as a pancreas would to control blood glucose levels. In this study, there are two types of VC-01 units that will be implanted. One is a larger VC-01 unit (VC-01-250) that is slightly smaller than the size of a business card, and the other is a smaller VC-01 unit (VC-01-20), that is about the size of a fingernail. Both products have the thickness of a credit card. While doctors and researchers believe that VC-01 might be an effective and convenient way for people to treat their type 1 diabetes, very few subjects have received implants, and risks are not yet known. It is an investigational combination product and is not yet approved for public use. Only men and women of non-childbearing potential who participate in this study will have the VC-01 combination product implanted in their bodies. In order to be approved for public use, governmental regulatory agencies require VC-01 to be evaluated in studies like this one. This study will help determine if VC-01 is a safe and effective treatment option for people with type 1 diabetes. If you have any questions about the VC-01 combination product, please ask the study doctor or study staff. WHAT WILL HAPPEN DURING THIS STUDY? If you participate in the study, you will be assigned to receive a select number of VC-01 units implanted during the study. Your smaller VC-01 units will be removed at various points in the study, but the larger units will not be removed until the end of the study, or sooner if you withdraw from the study early or if the Sponsor stops the study early. This study will last approximately 2 years and includes about 19 visits to the study clinic (including initial screening visits). Letter of Interest Date: 29 April 2015 Page 4 of 5 You may be asked to return for unplanned visits at any time for your safety. During your study clinic visits, doctors and staff members will conduct various tests and assessments to evaluate your response to the investigational product and overall health. Because the VC-01 units may take several months to begin working, you will continue your current insulin therapy during the study, as needed. The study doctor will monitor your diabetes and adjust your insulin doses when necessary to ensure your diabetes is appropriately treated for the entire study. You will be asked to closely monitor your blood glucose levels throughout the study. You will be provided with a blood glucose meter and supplies. Additionally, you would be provided with a continuous glucose monitoring (CGM) system that must be worn for at least 30 days before each study clinic visit. The study staff will also provide you with an electronic tablet diary so that you can record daily information about your insulin doses, hypoglycemia events, and fingerstick values from the blood glucose meter. A staff member will show you how to use these materials at Visit 2. You will need to bring your blood glucose meter, CGM system and electronic tablet diary to each clinic visit. After your final clinic visit, you will be asked to take part in a 3-year follow-up study where your safety will be monitored. WHAT ARE THE RISKS RELATED TO THIS STUDY? Because this is the first-in-human study, and very few subjects have received implants, risks are not yet known. Potential surgical risks include pain, bleeding, bruising, collection of fluid under the skin, tenderness, redness, and infection where the incisions were made. There are also risks of hypoglycemia events, the investigational product being ineffective, immune or inflammatory reactions, as well as other potential risks your study doctor will discuss with you. Additional information is included in the study informed consent document. Because research studies can affect your health and safety, you will be closely monitored throughout the study. Researchers for this study were required to design a protocol, which is a plan that explains all study procedures in detail. The University of Alberta Research Ethics Board and Health Canada have reviewed this protocol for good clinical practice and require that it be followed exactly as written. If you experience a side effect during this study, you would let the study doctor or a study staff member know immediately. STUDY VISIT REMINDERS If you participate in this study, you will be asked to visit the study clinic on a regular basis. During these visits, you will undergo various medical examinations and evaluations to monitor your health and progress. For your clinic visits, please always remember to: Honestly answer any study-related questions your study doctor or study staff members ask Ask questions about anything you do not understand Enter information daily in your electronic tablet diary and bring the tablet to each visit Wear your CGM system for at least 30 days before your visit Bring your blood glucose meter and CGM to each visit Letter of Interest Date: 29 April 2015 Page 5 of 5 Be prepared to complete questionnaires about your diabetes treatment satisfaction and quality of life Be prepared to give blood and urine samples Report any side effects you experience between visits If you are unable to come to a study clinic visit, please contact the study staff as soon as possible so that they can reschedule your visit.