Questions About the Human Clinical Trials
- How long will the human clinical trials take?
- How many phases do these trials have?
- In which phase are the trials now?
- When did the Phase I human clinical trial investigating BCG vaccination in patients with type 1 diabetes begin?
- What happens in Phase I? How long is Phase I going to take?
- Are you still enrolling patients for Phase I?
- What will happen in Phase II?
- What will be the criteria for Phase II?
- What should I do to be considered for the next phases of the clinical trial?
- What should I include in my brief medical history?
- Will you enroll children?
- I have been diabetic for many years; do I qualify for this trial?
- How does the selection process for the Phase II trials work and when will I know if I am selected?
- If enrolled, how often do I have to go to Boston?
- I am from outside the US and BCG is a mandatory vaccine in my country, why do I have type 1 diabetes?
- What are the blood donations for?
- What is a non-diabetic control?
- Can I send my blood to your lab instead of going to Boston?
- How can I set up an appointment for a blood donation visit?
- How often should I donate blood?
- Are the blood donations part of Phase I?
- Can children also donate blood?
- How can I contribute to the research?
Questions and Answers about the Human Clinical Trials
- How long will the human clinical trials take?
We expect the clinical trials to take around 8-10 years, but this depends on many factors, such as the requirements of the Food and Drug Administration (FDA) and the availability of funding for the different phases of the trials. The first Phase I clinical trial should be completed in about 18 months.
- How many phases do these trials have?
There are usually 4 phases - Phase I, II, III and IV.
- In which phase are the trials now?
We are in Phase I now.
- When did the Phase I human clinical trial investigating BCG vaccination in patients with type 1 diabetes begin?
The first Phase I trial started in January 2008.
- What happens in Phase I? How long is Phase I going to take?
In the first Phase I human clinical trial, subjects that meet the selection criteria and have been enrolled for this phase receive either a small dose of BCG or placebo. We expect this trial to last approximately 18 months.
- Are you still enrolling patients for Phase I?
Unfortunately, we are not. We have already reached the required number of subjects needed for our Phase I trial.
- What will happen in Phase II?
During the first Phase I trial, the safety of BCG is being tested. We expect to be successful in this trial since BCG is a generic drug approved for other uses in humans. In Phase II studies, we will want to begin to test efficacy and see if the drug can lead to clinical improvements, such as patients needing less insulin.
- What will be the criteria for Phase II?
We do not yet know because this will depend on the results of the Phase I study and on FDA requirements.
- What should I do to be considered for the next phases of the clinical trial?
To be a volunteer for Phase II, e-mail us a brief medical history, which will be put into our database. Also, we are currently collecting blood from diabetic and non-diabetic volunteers. Coming in for the blood study can help us see in more detail if you have the traits that are required for additional enrollment groups that may arise as the study evolves.
- What should I include in my brief medical history?
Please include:- Your date of birth
- Age of onset of diabetes
- Any complications associated with your type 1 diabetes
- Other major diseases that you might have (especially any other types of autoimmune disease)
- Any surgeries you have had
Also include your complete contact information in case we wish to reach you. Please include your name, address, e-mail address, and home/work/cell phone numbers.
- Will you enroll children?
We are not allowed to enroll minors in the Phase I trial, which is designed to prove the safety of the drug. However, we believe that at some point during the next phases, money permitting, we will enroll children, although this will depend on the FDA and on funding.
- I have been diabetic for many years; do I qualify for this trial?
Yes, these trials are enrolling patients that already have the disease.
- How does the selection process for the Phase II trials work and when will I know if I am selected?
Patients from our database that fit the selection criteria for enrollment in Phase II will be chosen. If you are selected, we will contact you.
- If enrolled, how often do I have to go to Boston?
The parameters for Phase II have not been fully designed yet, so we do not know. Currently, for the Phase I trial, patients have their blood drawn on a weekly basis for a period of 12 weeks.
- I am from outside the US and BCG is a mandatory vaccine in my country; why do I have type 1 diabetes?
You received BCG just once at a small dose as a vaccine to prevent tuberculosis. However, you most likely received this small dose prior to developing type 1 diabetes.
- What are the blood donations for?
The blood donations help us in the development of a blood test that monitors the treatment with BCG. In this test, we will be comparing your T-cells with the T-cells of a non-diabetic control. This blood donation also helps us to see if you might qualify for future studies, including later phases of the BCG interventional study.
- What is a non-diabetic control?
A non-diabetic control is a person who is not related to the patient by blood and does not have type 1 or type 2 diabetes. Also, the person should not have any other autoimmune diseases such as hypothyroidism, Crohn's disease, multiple sclerosis, or psoriasis.
- Can I send my blood to your lab instead of going to Boston?
Since we have to start analyzing the blood shortly after it is drawn, the blood donation has to be done in our lab in Boston, Mass.
- How can I set up an appointment for a blood donation visit?
You may either e-mail us at diabetestrial@partners.org or call the lab at 617-726-4084. Currently, we are booked a year in advance.
- How often should I donate blood?
The frequency of the blood drawing depends entirely on you. When coming in from out-of-town, you might want to schedule your visit for 2-3 days in a row so that we can draw 2-3 separate samples from you and thus make your visit cost effective.
- Are the blood donations part of Phase I?
The blood donations are voluntary and are used to help us further develop a blood assay used to monitor the treatment. Donating blood is not a requirement to be enrolled in our trials, but it may increase your chance of qualifying for future enrollments, since we will know if your blood might meet the needs for upcoming studies. If you come for a blood donation visit, you will not receive BCG or a placebo at that time. You will receive BCG or placebo only if you are selected to participate in the Phase I or Phase II human clinical trials.
- Can children also donate blood?
Patients 8 years of age and older can donate blood for our research.
- How can I contribute to the research?
You can contribute by scheduling a visit with us to provide a small sample of blood for our research. E-mail the lab to schedule a visit.
You can also make a gift online to support our work using the Massachusetts General Hospital's online gift giving form. Please note that your gift will be processed by Mass General's web site and that the "Other" field will read "Cure Diabetes Now" indicating your gift is in support of the Faustman Lab.
