Dear Individuals with Dystrophic EB,
I hope you are well. I am reaching out to you with a research study opportunity that might help push EB research forward. As you may be aware, Shire Pharmaceuticals is researching Investigational drugs that we all hope someday will treat the symptoms of dystrophic EB. Researching Investigational drugs is a complicated process that entails many stages. This letter will briefly outline what would be expected of you and how you will interact with the researchers if you participate.
In this stage of the project, Shire has engaged, Evidera, a contract research organization, to conduct interviews with people with dystrophic EB, as well as, their parents /caregivers. The idea is to develop a quality of life questionnaire. If you agree to participate, representatives from Evidera will contact you to discuss symptoms you, your child or patient experience and the impacts that are relevant to dystrophic EB. The goal is to make sure that the final questionnaires, which may be used in the clinical trials, accurately measure symptoms of dystrophic EB. The Jackson Gabriel Silver Foundation is helping Evidera to recruit participants for this stage of the research. What is important for you to know is that the Jackson Gabriel Silver Foundation and all of its representatives will not be directly involved in any of the interviews, nor will we have access to any of your answers. Evidera is required to make sure that all of your answers are confidential. Your interview would be audio recorded and a transcription would be made. If you do not wish to be recorded, you will not be able to participate in this study. All transcripts and any questionnaires you fill out would have a unique participant number and not have your actual name. Additionally, the audio recording and may be shared with Shire and/or Shire may listen during your actual interview. You would be informed if that were to case.
If you choose to be involved in this project, you will be asked questions about living with dystrophic EB and about the general burden of living with the disorder. Your participation is absolutely voluntary and you will have the right to withdraw from the study at any time. As a token of appreciation for your time, Evidera will provide the parent/caregiver or dystrophic EB patient a $150 American Express Gift Check. To be clear of any confusion, it would be a total of $150 (not $150 each).
Here is what you can expect from participating in the study;
· Evidera staff will call you to make sure you are eligible to participate. If eligible, Evidera will schedule a two hour interview at a time that is convenient for you;
· A couple of weeks after the first interview, Evidera will send you a few documents to review and conduct a brief (15-20 minute) call to obtain your feedback on them;
· Evidera will call you 1-2 days prior to the interview, and follow-up call, to remind you of the date and time;
· Evidera trained scientific staff will interview you via telephone or face-to-face at a location that is convenient to you;
Before the interview starts Evidera will first review the consent form with you to make sure you have no questions or concerns, and if you still want to participate you can sign the form at that time.
· If it is a telephone interview Evidera will send you a packet of information that will include informed consent forms to be completed before the interview starts and a sealed envelope, containing questionnaires that you will open and complete during the scheduled telephone interview;
· If it is a face-to-face interview Evidera will provide you with informed consents and assent forms prior to the start of the interview. There will be time to ask questions and receive answers prior to deciding whether or not you would like to participate. Questionnaires will be completed during the interview.
If you agree to participate, we will send your name and contact information (telephone number, email address and mailing address) to Evidera. Please let Geri Kelly EB Nurse Educator know by phone (866-332-7276) or email ([email protected]) if you give us permission to share this information.
Thank you for helping in our efforts to push the needle forward on dystrophic EB research.
Sincerely, Jamie Silver Jackson Gabriel Silver Foundation [email protected]
To learn more about the University of Minnesota trial, please click here.
Drs. Al Lane and Paul Khavari have labored over gene therapy to treat EB for years. After many years of work, Stanford hopes to commence a human trial soon. To learn more about Stanford’s gene therapy trial, please click here. A short video can be viewed below about gene therapy at Stanford.