Hydroxychloroquine (Plaquenil) Starts a Phase-II? Trial To Prevent Type-1 In Presymptomatics

This study is testing a medication, called hydroxychloroquine (HCQ) to assess safety and effectiveness to prevent individuals at risk of type 1 diabetes from progressing to type 1 diabetes.

HCQ is approved by the U.S. Food and Drug Administration as a treatment (not a cure) for malaria, lupus, and rheumatoid arthritis.  It operates through several pathways, including as an anti-inflammatory.  HCQ has been used for over 60 years, but has not previously been studied as a treatment to prevent T1D.

This Study

These researchers are recruiting 200 people who are at least 3 years old and test positive for 2 or more autoantibodies as part of TrialNet.  2/3s will get the drug and 1/3 will get a placebo and will be a control group.   The study involves 5 visits in the first 6 months, then 1 visit every 6 months for the remainder of the study (about six years).

The primary end point is the number of people who progress to having abnormal blood glucose numbers or to being diagnosed with type-1 diabetes.  (The hope is that few people getting the treatment will make this progression, as compared to the placebo group.)  The study started in Aug-2018 and they hope to finish in 2024.  Prevention studies take a long time, because the onset of type-1 takes a long time, so the researchers need to wait a long time to see if fewer people get the disease.

This trial is part of TrialNet and is being funded by JDRF.

The study's web page is here: https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq
This study is recruiting at several sites all over the US:

Barbara Davis Center: Aurora, Colorado, United States, 80045
Contact: Betsy Burke    303-724-6766    Betsy.Burke@ucdenver.edu 
Principal Investigator: Andrea Steck, MD
       
University of Florida: Gainesville, Florida, United States, 32610
Contact: Jennifer Hosford    352-294-5759    jennifer.hosford@medicine.ufl.edu 
Contact: Paula Towe    352-294-5761    pdtowe@peds.ufl.edu 
Principal Investigator: Desmond Schatz, MD   
  
University of Miami: Miami, Florida, United States, 33136
Contact: Della Matheson    305-243-3781    dmatheso@med.miami.edu 
Contact: Natalia Sanders-Branca    305-243-6616    n.sanders1@med.miami.edu 
Principal Investigator: David Baidal, MD 
    
University of Pittsburgh: Pittsburgh, Pennsylvania, United States, 15224
Contact: David Groscost    412-692-7241    david.groscost@chp.edu 
Contact: Kelli Delallo    412-692-5210    kelli.delallo@chp.edu 
Principal Investigator: Dorothy Becker, MD       

Vanderbilt Eskind Diabetes Clinic: Nashville, Tennessee, United States, 37232
Contact: Brenna Dixon    615-337-9597    brenna.dixon@vanderbilt.edu 
Contact: Faith Brendle    615-936-8638    faith.brendle@vanderbilt.edu 
Principal Investigator: William Russell, MD       

University of Texas Southwestern Recruiting
Dallas, Texas, United States, 75390
Contact: Lauren Boyles    241-648-4717    lauren.boyles@utsouthwestern.edu 
Principal Investigator: Philip Raskin, MD       

Benaroya Research Institute: Seattle, Washington, United States, 98101
Contact: Mary Ramey    206-342-6945    mramey@benaroyaresearch.org 
Contact: Marli McCulloch-Olson    206-515-5239    Marli@benaroyaresearch.org 
Principal Investigator: Carla Greenbaum, MD       

Wikipedia on HCQ: https://en.wikipedia.org/wiki/Hydroxychloroquine
Clinical Trial Registry:  https://clinicaltrials.gov/ct2/show/NCT03428945

Case studies:
* https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4206615/
* https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3029247
Use in Type-2:
* https://www.researchgate.net/publication/270282245_Ipca_gets_first_approval_for_HCQ_in_diabetes

Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.