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Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus (TN-22)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03428945
Recruitment Status : Recruiting
First Posted : February 12, 2018
Last Update Posted : August 15, 2019
Juvenile Diabetes Research Foundation
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

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.

Condition or disease Intervention/treatment Phase
Type1 Diabetes Mellitus Drug: Hydroxychloroquine Drug: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 201 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Treatment assignment (active drug: placebo) will be assigned in a parallel, randomized, 2:1 model. Assignment will be stratified based on prior treatment for T1D prevention and age.
Masking: Double (Participant, Investigator)
Masking Description: Active drug and placebo will be identical in appearance and packaging
Primary Purpose: Prevention
Official Title: Hydroxychloroquine for Prevention of Abnormal Glucose Tolerance and Diabetes in Individuals At-risk for Type 1 Diabetes Mellitus (T1D)
Actual Study Start Date : August 15, 2018
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : August 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Experimental: Hydroxychloroquine
Hydroxychloroquine compound for oral use
Drug: Hydroxychloroquine
Hydroxychloroquine for oral administration, dosed by weight
Other Name: Plaquenil

Placebo Comparator: Placebo
Placebo tablet matching active drug
Drug: Placebo
Placebo tablet identical to active drug

Primary Outcome Measures :
  1. Change from Treatment Assignment Glucose Tolerance to Abnormal Glucose Tolerance or Diabetes [ Time Frame: Glucose tolerance is measured every 6 months for approximately 6 years ]
    The primary outcome is the time from treatment assignment to changes in glucose tolerance. The time to development of abnormal glucose tolerance or the diagnosis of type 1 diabetes will be measured to identify these changes.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Participant in TrialNet Pathway to Prevention Study (TN01)
  2. Age 3 years or greater at the time of randomization
  3. Willing to provide informed consent
  4. Normal glucose tolerance by OGTT within 7 weeks (no more than 52 days) of baseline
  5. Two or more diabetes-related autoantibodies present on two separate samples
  6. Weight of 12 kg or greater at screening
  7. If a female participant with reproductive potential, willing to avoid pregnancy and undergo pregnancy testing prior to randomization and at each study visit
  8. Anticipated ability to swallow study medication.

Exclusion Criteria:

  1. Abnormal Glucose Tolerance or Diabetes
  2. History of treatment with insulin or other diabetes therapies
  3. Ongoing use of medications known to influence glucose tolerance
  4. Ongoing or anticipated future use of medications known to have untoward interactions with hydroxychloroquine
  5. Known hypersensitivity to 4-aminoquinoline compounds
  6. G6PD deficiency
  7. History of retinopathy
  8. Have an active infection at time of randomization
  9. Have serologic evidence of current or past HIV, Hepatitis B (positive for Hepatitis B core antibody or surface antigen), or Hepatitis C infection
  10. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03428945

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Contact: Lisa Rafkin 305-243-6146
Contact: Ryan O'Donnell 813-396-9551 Ryan.O'

  Hide Study Locations
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United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Daniel Bisno    323-361-8641   
Principal Investigator: Roshanak Monzavi, MD         
University of California - San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Rebecca Wesch    415-476-5984   
Contact: Karen Ko    415-514-3730   
Principal Investigator: Stephen Gitelman, MD         
Stanford University Recruiting
Stanford, California, United States, 94305
Contact: Trudy Esrey    650-498-4450   
Principal Investigator: Darrell Wilson, MD         
United States, Colorado
Barbara Davis Center Recruiting
Aurora, Colorado, United States, 80045
Contact: Betsy Burke    303-724-6766   
Principal Investigator: Andrea Steck, MD         
United States, Connecticut
Yale University School of Medicine Recruiting
New Haven, Connecticut, United States, 06519
Contact: Laurie Feldman    203-737-2760   
Principal Investigator: Kevan Herold, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Jennifer Hosford    352-294-5759   
Contact: Paula Towe    352-294-5761   
Principal Investigator: Desmond Schatz, MD         
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Della Matheson    305-243-3781   
Contact: Natalia Sanders-Branca    305-243-6616   
Principal Investigator: David Baidal, MD         
USF Diabetes Center Recruiting
Tampa, Florida, United States, 33612
Contact: Emily Eyth    813-974-9817   
Principal Investigator: Henry Rodriguez, MD         
United States, Georgia
Emory Children's Center Recruiting
Atlanta, Georgia, United States, 30322
Contact: Margaret Jenkins    404-712-0051   
Principal Investigator: Andrew Muir, MD         
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Gail Gannon    773-702-3853   
Principal Investigator: Louis Philipson, MD         
United States, Indiana
Indiana University - Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Maria Spall    317-278-8879   
Principal Investigator: Linda DiMeglio, MD         
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Joanne Cabbage    319-335-7434   
Principal Investigator: Michael Tansey, MD         
United States, Kentucky
University of Kentucky/UK Healthcare Recruiting
Lexington, Kentucky, United States, 40504
Contact: Kathy Edwards    859-218-6717   
Principal Investigator: Kathryn Thrailkill, MD         
University of Louisville Pediatric Endocrinology Recruiting
Louisville, Kentucky, United States, 40202
Contact: Stephany Eubanks    502-588-0491   
Principal Investigator: Kupper Wintergerst, MD         
United States, Massachusetts
Joslin Diabetes Center Recruiting
Boston, Massachusetts, United States, 02215
Contact: Nora Bryant    617-309-4141   
Principal Investigator: Jason Gaglia, MD         
United States, Michigan
University of Michigan Health System Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Andrea Haddad    734-615-4079   
Principal Investigator: Inas Thomas, MD         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Darcy Weingartner    612-624-5958   
Contact: Jessica Ruedy    612-624-6617   
Principal Investigator: Toni Moran, MD         
United States, Missouri
The Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
Contact: Lacey Deardoff    816-960-8943   
Principal Investigator: Wayne Moore, MD         
United States, Nevada
Palm Research Center, Inc. Recruiting
Las Vegas, Nevada, United States, 89148
Contact: Natasha Sushenko    702-736-5161   
Principal Investigator: Samer Nakhle, MD         
United States, New York
UBMD Pediatrics Recruiting
Buffalo, New York, United States, 14203
Contact: Amanda House    716-323-0075   
Principal Investigator: Teresa Quattrin, MD         
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Sarah Pollak    212-851-5465   
Contact: Analia Alvarez    212-851-5465   
Principal Investigator: Robin Goland, MD         
Joslin Center at SUNY Upstate Medical University Recruiting
Syracuse, New York, United States, 13210
Contact: Lynn Agostini    315-464-9012   
Principal Investigator: Ramachandra Naik, MD         
United States, Oklahoma
The University of Oklahoma Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact: Linda Weber    405-271-8001 ext 43074   
Principal Investigator: David Sparling, MD         
United States, Pennsylvania
Childrens Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104-4399
Contact: Beth Schwartzman    267-426-5718   
Principal Investigator: Lorraine Levitt Katz, MD         
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: David Groscost    412-692-7241   
Contact: Kelli Delallo    412-692-5210   
Principal Investigator: Dorothy Becker, MD         
United States, South Carolina
Endocrinology Specialist / Greenville Health System Recruiting
Greenville, South Carolina, United States, 29605
Contact: Shirley Parker    864-455-3261   
Principal Investigator: Sandra Weber, MD         
GHS - Pediatric Endocrinology Recruiting
Greenville, South Carolina, United States, 29615
Contact: Lisa Looper    864-454-5168   
Principal Investigator: Elaine Apperson, MD         
United States, Tennessee
Vanderbilt Eskind Diabetes Clinic Recruiting
Nashville, Tennessee, United States, 37232
Contact: Brenna Dixon    615-337-9597   
Contact: Faith Brendle    615-936-8638   
Principal Investigator: William Russell, MD         
United States, Texas
University of Texas Southwestern Recruiting
Dallas, Texas, United States, 75390
Contact: Lauren Boyles    241-648-4717   
Principal Investigator: Philip Raskin, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Hillarie Slater    801-585-6227   
Principal Investigator: Carol Foster, MD         
United States, Washington
Benaroya Research Institute Recruiting
Seattle, Washington, United States, 98101
Contact: Mary Ramey    206-342-6945   
Contact: Marli McCulloch-Olson    206-515-5239   
Principal Investigator: Carla Greenbaum, MD         
Australia, Victoria
Walter and Eliza Hall Institute of Medical Research Recruiting
Parkville, Victoria, Australia, 3052
Contact: Leanne Redl    61 3 9342 7063   
Principal Investigator: Peter Colman, MD         
Principal Investigator: John Wentworth, MD         
United Kingdom
University of Bristol Recruiting
Bristol, UK, United Kingdom, BS10 5NB
Contact: Beth Thorne    0117 4147917   
Principal Investigator: Polly Bingley, MD         
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Juvenile Diabetes Research Foundation
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Study Chair: Carla Greenbaum, MD Type 1 Diabetes TrialNet

Additional Information:
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Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Identifier: NCT03428945     History of Changes
Other Study ID Numbers: Hydroxychloroquine
UC4DK117009 ( U.S. NIH Grant/Contract )
UC4DK106993 ( U.S. NIH Grant/Contract )
First Posted: February 12, 2018    Key Record Dates
Last Update Posted: August 15, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antirheumatic Agents