Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus

Phase 2

Terminated

• Conditions: Type1 Diabetes Mellitus
• Interventions: Drug: Placebo; Drug: Hydroxychloroquine

• Registration Number: NCT03428945
• Lead Sponsor: 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.

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.

Study Timeline

• Study First Submitted: 2018-01-24
• Study First Submitted QC: 2018-02-08
• Study First Posted: 2018-02-12
• Study Start: 2018-08-15
• Primary Completion: 2022-10-27
• Study Completion: 2022-10-31
• Status Verified: 2023-07
• Results First Submitted: 2023-12-14
• Results First Submitted QC: 2024-03-13
• Last Update Submitted: 2024-03-13
• Results First Posted: 2024-04-09
• Last Update Posted: 2024-04-09

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 273

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.

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.

Trial Locations

Locations (51)

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

University of California - San Francisco; 🇺🇸 San Francisco, California, United States

Stanford University; 🇺🇸 Stanford, California, United States

Barbara Davis Center; 🇺🇸 Aurora, Colorado, United States

Yale University School of Medicine; 🇺🇸 New Haven, Connecticut, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of Miami; 🇺🇸 Miami, Florida, United States

USF Diabetes Center; 🇺🇸 Tampa, Florida, United States

Emory Children's Center; 🇺🇸 Atlanta, Georgia, United States

St. Luke's Humphreys Diabetes Center; 🇺🇸 Boise, Idaho, United States

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