Back to resultsHydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus (TN-22)
Primary Purpose
Type1 Diabetes Mellitus
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Hydroxychloroquine
Placebo
Sponsored by
About this trial
This is an interventional prevention trial for Type1 Diabetes Mellitus focused on measuring TrialNet
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Sites / Locations
- Children's Hospital Los Angeles
- University of California - San Francisco
- Stanford University
- Barbara Davis Center
- Yale University School of Medicine
- University of Florida
- University of Miami
- USF Diabetes Center
- Emory Children's Center
- St. Luke's Humphreys Diabetes Center
- Rocky Mountain Clinical Research
- University of Chicago
- Indiana University - Riley Hospital for Children
- University of Iowa
- University of Kentucky/UK Healthcare
- University of Louisville Pediatric Endocrinology
- Joslin Diabetes Center
- University of Michigan Health System
- University of Minnesota
- The Children's Mercy Hospital
- Palm Research Center, Inc.
- UBMD Pediatrics
- Columbia University
- Joslin Center at SUNY Upstate Medical University
- Cleveland Clinic Foundation
- The University of Oklahoma
- Childrens Hospital of Philadelphia
- University of Pittsburgh
- Endocrinology Specialist / Greenville Health System
- GHS - Pediatric Endocrinology
- Sanford Children's Specialty Clinic
- Vanderbilt Eskind Diabetes Clinic
- University of Texas Southwestern
- University of Utah
- University of Virginia Health System
- Benaroya Research Institute
- Medical College of Wisconsin
- Royal North Shore Hospital
- Women's and Children's Hospital, Adelaide
- Walter and Eliza Hall Institute of Medical Research
- Perth Children's Hospital
- Children's Hospital of Eastern Ontario
- The Hospital For Sick Kids
- IWK Health Center
- San Raffaele Hospital
- Skane University Hospital
- Royal Devon and Exeter Hospital
- Plymouth Diabetes Clinical Research Unit
- Harrogate and District NHS Foundation Trust
- University of Bristol
- Nottingham University Hospitals
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Hydroxychloroquine
Placebo
Arm Description
Hydroxychloroquine compound for oral use
Placebo tablet matching active drug
Outcomes
Primary Outcome Measures
Change from Treatment Assignment Glucose Tolerance to Abnormal Glucose Tolerance or Diabetes
The primary outcome is the time from treatment assignment to changes in glucose tolerance. The time to development of abnormal glucose tolerance or the diagnosis of type 1 diabetes will be measured to identify these changes.
Secondary Outcome Measures
Full Information
NCT ID
NCT03428945
First Posted
January 24, 2018
Last Updated
July 14, 2023
Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators
Juvenile Diabetes Research Foundation
1. Study Identification
Unique Protocol Identification Number
NCT03428945
Brief Title
Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus
Acronym
TN-22
Official Title
Hydroxychloroquine for Prevention of Abnormal Glucose Tolerance and Diabetes in Individuals At-risk for Type 1 Diabetes Mellitus (T1D)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
This trial has undergone a prespecified interim analysis which determined that this treatment provides no statistically significant delay in the onset of abnormal glucose tolerance or Type 1 Diabetes.
Study Start Date
August 15, 2018 (Actual)
Primary Completion Date
October 27, 2022 (Actual)
Study Completion Date
October 31, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators
Juvenile Diabetes Research Foundation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type1 Diabetes Mellitus
Keywords
TrialNet
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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.
Masking
ParticipantInvestigator
Masking Description
Active drug and placebo will be identical in appearance and packaging
Allocation
Randomized
Enrollment
275 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Hydroxychloroquine
Arm Type
Experimental
Arm Description
Hydroxychloroquine compound for oral use
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo tablet matching active drug
Intervention Type
Drug
Intervention Name(s)
Hydroxychloroquine
Other Intervention Name(s)
Plaquenil
Intervention Description
Hydroxychloroquine for oral administration, dosed by weight
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Inactive Drug
Intervention Description
Placebo tablet identical to active drug
Primary Outcome Measure Information:
Title
Change from Treatment Assignment Glucose Tolerance to Abnormal Glucose Tolerance or Diabetes
Description
The primary outcome is the time from treatment assignment to changes in glucose tolerance. The time to development of abnormal glucose tolerance or the diagnosis of type 1 diabetes will be measured to identify these changes.
Time Frame
Glucose tolerance is measured every 6 months for approximately 6 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carla Greenbaum, MD
Organizational Affiliation
Type 1 Diabetes TrialNet
Official's Role
Study Chair
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California - San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Barbara Davis Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Yale University School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
USF Diabetes Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Emory Children's Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
St. Luke's Humphreys Diabetes Center
City
Boise
State/Province
Idaho
ZIP/Postal Code
83702
Country
United States
Facility Name
Rocky Mountain Clinical Research
City
Idaho Falls
State/Province
Idaho
ZIP/Postal Code
83404
Country
United States
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Indiana University - Riley Hospital for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kentucky/UK Healthcare
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40504
Country
United States
Facility Name
University of Louisville Pediatric Endocrinology
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Joslin Diabetes Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
University of Michigan Health System
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Palm Research Center, Inc.
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89148
Country
United States
Facility Name
UBMD Pediatrics
City
Buffalo
State/Province
New York
ZIP/Postal Code
14203
Country
United States
Facility Name
Columbia University
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Joslin Center at SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
The University of Oklahoma
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Childrens Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104-4399
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Endocrinology Specialist / Greenville Health System
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29605
Country
United States
Facility Name
GHS - Pediatric Endocrinology
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29615
Country
United States
Facility Name
Sanford Children's Specialty Clinic
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57105
Country
United States
Facility Name
Vanderbilt Eskind Diabetes Clinic
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Virginia Health System
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Benaroya Research Institute
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Royal North Shore Hospital
City
St. Leonards
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
Women's and Children's Hospital, Adelaide
City
North Adelaide
State/Province
South Australia
ZIP/Postal Code
5006
Country
Australia
Facility Name
Walter and Eliza Hall Institute of Medical Research
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Perth Children's Hospital
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6840
Country
Australia
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H8L1
Country
Canada
Facility Name
The Hospital For Sick Kids
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G-1X8
Country
Canada
Facility Name
IWK Health Center
City
Halifax
ZIP/Postal Code
NS B3K 6R8
Country
Canada
Facility Name
San Raffaele Hospital
City
Milan
ZIP/Postal Code
20132
Country
Italy
Facility Name
Skane University Hospital
City
Malmö
ZIP/Postal Code
20502
Country
Sweden
Facility Name
Royal Devon and Exeter Hospital
City
Exeter
State/Province
Devon
ZIP/Postal Code
EX2 5DW
Country
United Kingdom
Facility Name
Plymouth Diabetes Clinical Research Unit
City
Plymouth
State/Province
Devon
ZIP/Postal Code
PLG8BX
Country
United Kingdom
Facility Name
Harrogate and District NHS Foundation Trust
City
Harrogate
State/Province
North Yorkshire
ZIP/Postal Code
HG2 7SX
Country
United Kingdom
Facility Name
University of Bristol
City
Bristol
State/Province
UK
ZIP/Postal Code
BS10 5NB
Country
United Kingdom
Facility Name
Nottingham University Hospitals
City
Nottingham
ZIP/Postal Code
NG7 2UH
Country
United Kingdom
12. IPD Sharing Statement
Links:
URL
http://www.trialnet.org/
Description
TrialNet Public Website
Learn more about this trial
Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus
We'll reach out to this number within 24 hrs