Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus

NCT03428945 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: HydroxychloroquineUC4DK117009UC4DK106993
• Study Type: interventional
• Target: 273
• Locations: 6 countries
• Sites: 51

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.

Conditions

Type1 Diabetes Mellitus

Keywords

TrialNet

Outcome Measures

Primary Outcomes (1)

• Change From Treatment Assignment Glucose Tolerance to Abnormal Glucose Tolerance or Diabetes

  The primary outcome is the time in months from random treatment assignment to the development of confirmed abnormal glucose tolerance or clinical diabetes.

  Glucose tolerance is measured every 6 months for up to 4 years

Study Arms (2)

Hydroxychloroquine

EXPERIMENTAL

Hydroxychloroquine compound for oral use

Drug: Hydroxychloroquine

Placebo

PLACEBO COMPARATOR

Placebo tablet matching active drug

Drug: Placebo

Interventions

Hydroxychloroquine DRUG

Hydroxychloroquine for oral administration, dosed by weight

Also known as: Plaquenil

Hydroxychloroquine

Placebo DRUG

Placebo tablet identical to active drug

Also known as: Inactive Drug

Placebo

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead
• Juvenile Diabetes Research Foundation: collaborator

Study Sites (51)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of California - San Francisco

San Francisco, California, 94143, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

Barbara Davis Center

Aurora, Colorado, 80045, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

USF Diabetes Center

Tampa, Florida, 33612, United States

Location

Emory Children's Center

Atlanta, Georgia, 30322, United States

Location

St. Luke's Humphreys Diabetes Center

Boise, Idaho, 83702, United States

Location

Rocky Mountain Clinical Research

Idaho Falls, Idaho, 83404, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Indiana University - Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kentucky/UK Healthcare

Lexington, Kentucky, 40504, United States

Location

University of Louisville Pediatric Endocrinology

Louisville, Kentucky, 40202, United States

Location

Joslin Diabetes Center

Boston, Massachusetts, 02215, United States

Location

University of Michigan Health System

Ann Arbor, Michigan, 48109, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Palm Research Center, Inc.

Las Vegas, Nevada, 89148, United States

Location

UBMD Pediatrics

Buffalo, New York, 14203, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Joslin Center at SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

The University of Oklahoma

Oklahoma City, Oklahoma, 73104, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-4399, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Endocrinology Specialist / Greenville Health System

Greenville, South Carolina, 29605, United States

Location

GHS - Pediatric Endocrinology

Greenville, South Carolina, 29615, United States

Location

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, 57105, United States

Location

Vanderbilt Eskind Diabetes Clinic

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

Benaroya Research Institute

Seattle, Washington, 98101, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Royal North Shore Hospital

St Leonards, New South Wales, 2065, Australia

Location

Women's and Children's Hospital, Adelaide

North Adelaide, South Australia, 5006, Australia

Location

Walter and Eliza Hall Institute of Medical Research

Parkville, Victoria, 3052, Australia

Location

Perth Children's Hospital

Perth, Western Australia, 6840, Australia

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada

Location

The Hospital For Sick Kids

Toronto, Ontario, M5G-1X8, Canada

Location

IWK Health Center

Halifax, NS B3K 6R8, Canada

Location

San Raffaele Hospital

Milan, 20132, Italy

Location

Skane University Hospital

Malmo, 20502, Sweden

Location

Royal Devon and Exeter Hospital

Exeter, Devon, EX2 5DW, United Kingdom

Location

Plymouth Diabetes Clinical Research Unit

Plymouth, Devon, PLG8BX, United Kingdom

Location

Harrogate and District NHS Foundation Trust

Harrogate, North Yorkshire, HG2 7SX, United Kingdom

Location

University of Bristol

Bristol, UK, BS10 5NB, United Kingdom

Location

Nottingham University Hospitals

Nottingham, NG7 2UH, United Kingdom

Location

Related Links

• TrialNet Public Website

MeSH Terms

Interventions

Hydroxychloroquine

Intervention Hierarchy (Ancestors)

Chloroquine; Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Limitations and Caveats

Interim analysis completed prompted the DSMB's decision to terminate the protocol early limiting the originally planned follow up period for participants.

Results Point of Contact

• Title: Dr. Kevan Herold
• Organization: Yale University

Kevan.Herold@yale.edu

203-785-6507

Study Officials

• Carla Greenbaum, MD

  Type 1 Diabetes TrialNet

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Active drug and placebo will be identical in appearance and packaging
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 24, 2018
• First Posted: February 12, 2018
• Study Start: August 15, 2018
• Primary Completion: October 27, 2022
• Study Completion: October 31, 2022
• Last Updated: April 9, 2024
• Results First Posted: April 9, 2024

Record last verified: 2023-07

Locations

US (37); CA (3); IT (1); SE (1); AU (4); GB (5)