We are testing the drug hydroxychloroquine (HCQ) to see if it can delay or prevent early stage T1D (stage 1) from progressing to abnormal glucose tolerance (stage 2) and ultimately prevent clinical diagnosis (stage 3). HCQ is already used to reduce symptoms and progression of other autoimmune diseases, such as rheumatoid arthritis and lupus. This is the first study to see if it can prevent or delay T1D.
Hydroxychloroquine (HCQ) is approved by the Food and Drug Administration (FDA) and has been used for more than 60 years. It is used to treat other autoimmune diseases, including rheumatoid arthritis and lupus. HCQ has a good safety profile and is approved for use in children.
As with any medical intervention, there are risks and benefits to participating in this study. Before you decide to participate, a member our research team will explain all potential risks and benefits, and answer any questions you may have.
To find out if this study is right for you, the first step is to sign up for Pathway to Prevention screening. To participate in the HCQ Prevention Study, you need to:
- be at least 3 years old
- have two or more diabetes-related autoantibodies (confirmed by TrialNet screening in last 6 months)
- have normal results on an oral glucose tolerance test (OGTT) within 7 weeks of starting the study
- be able to swallow a whole capsule
- NOT be pregnant or planning to become pregnant while in the study
- NOT have any medical conditions that might make it unsafe for you to be in this study
You will take the study drug until you progress to stage 2 T1D (abnormal glucose tolerance) or stage 3 (diabetes onset). Your initial study visit will be followed by visits at months 3 and 6, and then every 6 months.
While taking the study medication, you will also need to undergo a study-specific eye exam within 3 months of starting the study drug, and then once a year while taking study medication.
If you progress to stage 2 diabetes (abnormal glucose tolerance), you will stop taking study drug, but you may choose to continue to visit a study site every six months for monitoring until the study ends.
Study visit 1
At your first study visit, you will be randomly placed into one of two groups. One group will get the study drug (HCQ). The other group will get a placebo (looks like the study drug but is inactive). You will have a two out of three chance of getting HCQ. A computer randomly selects who gets the study drug. No one knows who is in which group until the end of the study.
You will receive a 3-month supply of the study drug to take as instructed until your next visit.
Study visit 2
At the 3-month visit, you will have blood tests and monitoring. You will also receive another 3-month supply of the study drug to take as instructed.
Within 3 months of starting the study drug, you will also need to undergo a study-specific eye exam performed by an optometrist or ophthalmologist. Your study site will inform you where to go to have the eye exams completed (at no cost to you).
Study visit 3
At the 6-month visit, in addition to blood tests and monitoring, you will undergo an OGTT. You will receive a 6-month supply of the study drug to take as instructed.
Study visit 4 and forward (every six months)
You will visit a study site every 6 months for blood tests and monitoring. At each visit, you will receive a 6-month supply of the study drug to take as instructed until your next visit.
While taking the study drug, you will need to undergo a study-specific eye exam performed by an optometrist or ophthalmologist once a year. Your study site will inform you where to go to have the eye exams completed (at no cost to you).
Benefits of monitoring
All study participants receive close health monitoring. If you develop abnormal glucose tolerance, you can continue in this study, but will no longer take study medication. If eligible, you may join another prevention study.
If you develop T1D, our tests will likely show this before you have any symptoms. For people taking part in T1D research, the risk of diabetic ketoacidosis (DKA) at diagnosis drops from 30% to less than 3%. DKA is a serious condition that can be life-threatening. With early notification, you will be able to see your doctor and start taking insulin before you feel sick.
If diagnosed with T1D, you can no longer be in this study, but you may be able to join a study for people with newly diagnosed T1D or the TrialNet Long-term Follow-Up Study (LIFT).