Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus
- Conditions
- Type1 Diabetes Mellitus
- Interventions
- Drug: Placebo
- Registration Number
- NCT03428945
- Brief Summary
The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo controlled clinical trial. Subjects will receive hydroxychloroquine or placebo and close monitoring for progression of T1D.
- Detailed Description
This study is testing a medication, called hydroxychloroquine (HCQ) to assess safety and effectiveness to prevent individuals at risk of type 1 diabetes (T1D) from progressing to type 1 diabetes.
HCQ is approved by the U.S. Food and Drug Administration as a treatment for malaria, lupus, and rheumatoid arthritis. HCQ has been used extensively for treatment of autoimmune disease in adults, children, and during pregnancy. This medication has not previously been studied as a treatment to prevent T1D.
The goal of this study is to learn if HCQ can help prevent or delay progression from normal glucose tolerance (Stage 1) to abnormal glucose tolerance (Stage 2) or type 1 diabetes (Stage 3).
The study involves 5 visits in the first 6 months, then 1 visit every 6 months for the remainder of the study.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 273
- Participant in TrialNet Pathway to Prevention Study (TN01)
- Age 3 years or greater at the time of randomization
- Willing to provide informed consent
- Normal glucose tolerance by OGTT within 7 weeks (no more than 52 days) of baseline
- Two or more diabetes-related autoantibodies present on two separate samples
- Weight of 12 kg or greater at screening
- If a female participant with reproductive potential, willing to avoid pregnancy and undergo pregnancy testing prior to randomization and at each study visit
- Anticipated ability to swallow study medication.
- Abnormal Glucose Tolerance or Diabetes
- History of treatment with insulin or other diabetes therapies
- Ongoing use of medications known to influence glucose tolerance
- Ongoing or anticipated future use of medications known to have untoward interactions with hydroxychloroquine
- Known hypersensitivity to 4-aminoquinoline compounds
- G6PD deficiency
- History of retinopathy
- Have an active infection at time of randomization
- Have serologic evidence of current or past HIV, Hepatitis B (positive for Hepatitis B core antibody or surface antigen), or Hepatitis C infection
- Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
- Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
- Be pregnant or breastfeeding.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Placebo tablet matching active drug Hydroxychloroquine Hydroxychloroquine Hydroxychloroquine compound for oral use
- Primary Outcome Measures
Name Time Method Change From Treatment Assignment Glucose Tolerance to Abnormal Glucose Tolerance or Diabetes Glucose tolerance is measured every 6 months for up to 4 years The primary outcome is the time in months from random treatment assignment to the development of confirmed abnormal glucose tolerance or clinical diabetes.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (51)
Skane University Hospital
🇸🇪Malmö, Sweden
Joslin Center at SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
Endocrinology Specialist / Greenville Health System
🇺🇸Greenville, South Carolina, United States
IWK Health Center
🇨🇦Halifax, Canada
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
UBMD Pediatrics
🇺🇸Buffalo, New York, United States
Stanford University
🇺🇸Stanford, California, United States
Yale University School of Medicine
🇺🇸New Haven, Connecticut, United States
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
St. Luke's Humphreys Diabetes Center
🇺🇸Boise, Idaho, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
Rocky Mountain Clinical Research
🇺🇸Idaho Falls, Idaho, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
The Hospital For Sick Kids
🇨🇦Toronto, Ontario, Canada
Columbia University
🇺🇸New York, New York, United States
University of Kentucky/UK Healthcare
🇺🇸Lexington, Kentucky, United States
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Nottingham University Hospitals
🇬🇧Nottingham, United Kingdom
Royal North Shore Hospital
🇦🇺St. Leonards, New South Wales, Australia
Women's and Children's Hospital, Adelaide
🇦🇺North Adelaide, South Australia, Australia
Plymouth Diabetes Clinical Research Unit
🇬🇧Plymouth, Devon, United Kingdom
University of Chicago
🇺🇸Chicago, Illinois, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
University of Miami
🇺🇸Miami, Florida, United States
Indiana University - Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Benaroya Research Institute
🇺🇸Seattle, Washington, United States
University of Louisville Pediatric Endocrinology
🇺🇸Louisville, Kentucky, United States
University of Michigan Health System
🇺🇸Ann Arbor, Michigan, United States
Childrens Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Bristol
🇬🇧Bristol, UK, United Kingdom
Palm Research Center, Inc.
🇺🇸Las Vegas, Nevada, United States
University of California - San Francisco
🇺🇸San Francisco, California, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Vanderbilt Eskind Diabetes Clinic
🇺🇸Nashville, Tennessee, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Perth Children's Hospital
🇦🇺Perth, Western Australia, Australia
Harrogate and District NHS Foundation Trust
🇬🇧Harrogate, North Yorkshire, United Kingdom
University of Florida
🇺🇸Gainesville, Florida, United States
San Raffaele Hospital
🇮🇹Milan, Italy
Barbara Davis Center
🇺🇸Aurora, Colorado, United States
USF Diabetes Center
🇺🇸Tampa, Florida, United States
The University of Oklahoma
🇺🇸Oklahoma City, Oklahoma, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
University of Texas Southwestern
🇺🇸Dallas, Texas, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
GHS - Pediatric Endocrinology
🇺🇸Greenville, South Carolina, United States
Sanford Children's Specialty Clinic
🇺🇸Sioux Falls, South Dakota, United States
Joslin Diabetes Center
🇺🇸Boston, Massachusetts, United States
Royal Devon and Exeter Hospital
🇬🇧Exeter, Devon, United Kingdom
Walter and Eliza Hall Institute of Medical Research
🇦🇺Parkville, Victoria, Australia