Print

Purpose

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

Eligibility

Eligible Ages
Over 3 Years
Eligible Genders
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.

Study Design

Phase
Phase 2
Study Type
Interventional
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.
Primary Purpose
Prevention
Masking
Double (Participant, Investigator)
Masking Description
Active drug and placebo will be identical in appearance and packaging

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Hydroxychloroquine
Hydroxychloroquine compound for oral use
  • Drug: Hydroxychloroquine
    Hydroxychloroquine for oral administration, dosed by weight
    Other names:
    • Plaquenil
Placebo Comparator
Placebo
Placebo tablet matching active drug
  • Drug: Placebo
    Placebo tablet identical to active drug
    Other names:
    • Inactive Drug

Recruiting Locations

UK Center for Clinical and Translational Science and nearby locations

University of Kentucky/UK Healthcare
Lexington, Kentucky 40504
Contact:
Kathy Edwards
859-218-6717
kathy.edwards@uky.edu

More Details

NCT ID
NCT03428945
Status
Recruiting
Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Contact

Lisa Rafkin
305-243-6146
lrafkin@miami.edu

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.