Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus

NCT ID: NCT03428945

Last Updated: 2024-04-09

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

273 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-08-15

Study Completion Date

2022-10-31

Brief Summary

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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

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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.

Conditions

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Type1 Diabetes Mellitus

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators
Active drug and placebo will be identical in appearance and packaging

Study Groups

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Hydroxychloroquine

Hydroxychloroquine compound for oral use

Group Type EXPERIMENTAL

Hydroxychloroquine

Intervention Type DRUG

Hydroxychloroquine for oral administration, dosed by weight

Placebo

Placebo tablet matching active drug

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo tablet identical to active drug

Interventions

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Hydroxychloroquine

Hydroxychloroquine for oral administration, dosed by weight

Intervention Type DRUG

Placebo

Placebo tablet identical to active drug

Intervention Type DRUG

Other Intervention Names

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Plaquenil Inactive Drug

Eligibility Criteria

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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.
11. 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.
12. Be pregnant or breastfeeding.
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Juvenile Diabetes Research Foundation

OTHER

Sponsor Role collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Carla Greenbaum, MD

Role: STUDY_CHAIR

Type 1 Diabetes TrialNet

Locations

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Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

University of California - San Francisco

San Francisco, California, United States

Site Status

Stanford University

Stanford, California, United States

Site Status

Barbara Davis Center

Aurora, Colorado, United States

Site Status

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

USF Diabetes Center

Tampa, Florida, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

St. Luke's Humphreys Diabetes Center

Boise, Idaho, United States

Site Status

Rocky Mountain Clinical Research

Idaho Falls, Idaho, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

Indiana University - Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

University of Kentucky/UK Healthcare

Lexington, Kentucky, United States

Site Status

University of Louisville Pediatric Endocrinology

Louisville, Kentucky, United States

Site Status

Joslin Diabetes Center

Boston, Massachusetts, United States

Site Status

University of Michigan Health System

Ann Arbor, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Palm Research Center, Inc.

Las Vegas, Nevada, United States

Site Status

UBMD Pediatrics

Buffalo, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Joslin Center at SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

The University of Oklahoma

Oklahoma City, Oklahoma, United States

Site Status

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Endocrinology Specialist / Greenville Health System

Greenville, South Carolina, United States

Site Status

GHS - Pediatric Endocrinology

Greenville, South Carolina, United States

Site Status

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, United States

Site Status

Vanderbilt Eskind Diabetes Clinic

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Benaroya Research Institute

Seattle, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Royal North Shore Hospital

St Leonards, New South Wales, Australia

Site Status

Women's and Children's Hospital, Adelaide

North Adelaide, South Australia, Australia

Site Status

Walter and Eliza Hall Institute of Medical Research

Parkville, Victoria, Australia

Site Status

Perth Children's Hospital

Perth, Western Australia, Australia

Site Status

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

The Hospital For Sick Kids

Toronto, Ontario, Canada

Site Status

IWK Health Center

Halifax, , Canada

Site Status

San Raffaele Hospital

Milan, , Italy

Site Status

Skane University Hospital

Malmo, , Sweden

Site Status

Royal Devon and Exeter Hospital

Exeter, Devon, United Kingdom

Site Status

Plymouth Diabetes Clinical Research Unit

Plymouth, Devon, United Kingdom

Site Status

Harrogate and District NHS Foundation Trust

Harrogate, North Yorkshire, United Kingdom

Site Status

University of Bristol

Bristol, UK, United Kingdom

Site Status

Nottingham University Hospitals

Nottingham, , United Kingdom

Site Status

Countries

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United States Australia Canada Italy Sweden United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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http://www.trialnet.org/

TrialNet Public Website

Other Identifiers

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UC4DK117009

Identifier Type: NIH

Identifier Source: secondary_id

View Link

UC4DK106993

Identifier Type: NIH

Identifier Source: secondary_id

View Link

Hydroxychloroquine

Identifier Type: -

Identifier Source: org_study_id

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