Denise Faustman, MD, PHd
The Faustman Lab at work

Support the search for a cure
The research conducted by the Faustman Lab depends on the support of people like you.
>Make a gift today!

Questions About the Human Clinical Trials

  1. How long will the human clinical trials take?
  2. How many phases do these trials have?
  3. In which phase are the trials now?
  4. When did the Phase I human clinical trial investigating BCG vaccination in patients with type 1 diabetes begin? When did Phase II begin?
  5. What happened in Phase I?
  6. Are you currently enrolling patients in clinical trials?
  7. What will happen in Phase II?
  8. What will be the criteria for Phase II?
  9. What should I do to be considered for the next phases of the clinical trial?
  10. Will you enroll children?
  11. I have been diabetic for many years; do I qualify for this trial?
  12. How does the selection process for the Phase II trials work and when will I know if I am selected?
  13. If enrolled, how often do I have to go to Boston?
  14. I am from outside the US and BCG is a mandatory vaccine in my country, why do I have type 1 diabetes?
  15. What are the blood donations for?
  16. What is a non-diabetic control?
  17. Can I send my blood to your lab instead of going to Boston?
  18. How can I set up an appointment for a blood donation visit?
  19. How often should I donate blood?
  20. Are the blood donations part of the human clinical studies?
  21. Can children also donate blood?
  22. How can I contribute to the research?

Questions and Answers about the Human Clinical Trials

  1. 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.

  2. How many phases do these trials have?
    There are up to four phases - Phase I, II, III and IV. For more information, please visit http://www.nlm.nih.gov/services/ctphases.html

  3. In which phase are the trials now?
    We are planning Phase II now.

  4. When did the Phase I human clinical trial investigating BCG vaccination in patients with type 1 diabetes begin? When did Phase II begin?
    The trial started in January 2008 and was completed in 2011. The start of the Phase II study was announced in June 2011.

  5. What happens in Phase I? How long is Phase I going to take?
    The FDA-approved, double-blind, placebo-controlled, Phase I human clinical trial investigated the safety of BCG vaccination in individuals living with type 1 diabetes. The trial showed that BCG vaccination is safe for type 1 diabetics.  There were no severe reactions to the vaccine. Mild inflammation was observed at the injection site, as expected. The U.S. Food and Drug Administration (FDA) and the MGH data safety monitoring board have confirmed that Phase I testing adequately showed that BCG was safe as used in diabetics in this study. Please read our press release here:

    http://www.eurekalert.org/pub_releases/2010-06/mgh-mgh062810.php

    Data on four outcome measures from the Phase I trial were presented at the American Diabetes Association's 71st Scientific Sessions in June 2011. The data showed that induction of tumor necrosis factor (TNF) in individuals with long-term diabetes appears to: selectively eliminate the autoreactive T cells that destroy the pancreas, increase the number of regulatory T cells (Tregs), and transiently restore insulin production as measured by C-peptide. Fluctuations in GAD autoantibodies were also observed, suggesting BCG was having a positive effect on the pancreas, i.e., islet cell regeneration may have been starting to occur. Please click here to read our Phase I Frequently Asked Questions. Please read the press release here:

    http://www.eurekalert.org/pub_releases/2011-06/mgh-mgh061711.php


  6. Are you currently enrolling patients in any clinical studies?
    Although we are not enrolling patients currently into the interventional studies, we are screening patients for Phase II and also enrolling patients daily into tissue culture studies examining their blood for TNF-sensitive immune cells to determine eligibility.

  7. What will happen in Phase II?
    In Phase II, we will try to identify the best dose of BCG and the optimal timing of BCG administration. This study will cost a total of $25.2 million over a three-year period. Please consider making a donation to support this research.

  8. What will be the criteria for Phase II?
    We do not yet know. It will depend on the data from the Phase I study and on FDA requirements. We are currently beginning the planning stages for this trial.

  9. What should I do to be considered for the next phases of the clinical trial?
    Please fill out a patient information form to be a volunteer for Phase II, which will be put into our database. You can send the form to us at diabetestrial@partners.org. Also, we are currently collecting blood from diabetic and non-diabetic volunteers to see if they have TNF-sensitive immune cells. Please make an appointment by emailing the above email address. 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.

  10. Will you enroll children?
    We are enrolling children for the blood donation studies that are ongoing if they are older than 8 years old. We do not yet know if children will be enrolled into Phase II testing at this time.

  11. I have been diabetic for many years; do I qualify for this trial?
    Yes, these trials are enrolling patients that already have the disease.

  12. 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.

  13. 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. For the Phase I trial, patients had their blood drawn on a weekly basis.

  14. 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. We are testing BCG at higher doses and at different administration schedules than those currently used for tuberculosis prevention.

  15. What are the blood donations for?
    The blood donations help us in the development of a blood test that monitors the treatment with BCG. This blood donation also helps us to see if you might qualify for future studies, including later phases of the BCG interventional study.

  16. 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.

  17. 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.

  18. 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.

  19. 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.

  20. Are the blood donations part of the human clinical studies?
    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 placebo at that time. You will receive BCG or placebo only if you are selected to participate in the human clinical trials.

  21. Can children also donate blood?
    Patients 8 years of age and older can donate blood for our research.

  22. 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.