NCT02603146

Brief Summary

The purpose of this study is to determine if hydroxychloroquine (HCQ) is safe and effective for the prevention of future onset of rheumatoid arthritis (RA) in individuals who have elevations of an autoantibody, anti-cyclic citrullinated peptide (anti-CCP3). The following recruitment strategies will be employed towards identifying healthy subjects with elevated anti-cyclic citrullinated peptide (anti-CCP3) levels:

  • Pre-screening:
  • first degree relatives of patients with rheumatoid arthritis (RA);
  • subjects at health-fairs; and
  • identification of subjects with elevated anti-CCP3 levels in the absence of inflammatory arthritis in rheumatology clinics.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
144

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Apr 2016

Longer than P75 for phase_2

Geographic Reach
1 country

14 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 10, 2015

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 11, 2015

Completed
6 months until next milestone

Study Start

First participant enrolled

April 27, 2016

Completed
6.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2022

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

November 21, 2023

Completed
Last Updated

June 12, 2025

Status Verified

May 1, 2025

Enrollment Period

6.5 years

First QC Date

November 10, 2015

Results QC Date

October 30, 2023

Last Update Submit

May 27, 2025

Conditions

Keywords

RA preventionhydroxychloroquine (HCQ)anti-CCP3

Outcome Measures

Primary Outcomes (1)

  • Number of Participants Who Developed Clinically-Apparent Rheumatoid Arthritis (CL - RA) From Treatment Initiation to Month 36 By Treatment Arm

    Clinically-Apparent RA is defined by the 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) Classification Criteria as either: 1.) a score of ≥ 6 defining "definite RA" or 2.) a joint examination consistent with Inflammatory Arthritis (IA) with ≥ 1 erosion confirmed by x-ray imaging of the hands, wrists, and feet.

    Baseline to Month 36

Secondary Outcomes (14)

  • Number of Participants Who Developed Clinically-Apparent Rheumatoid Arthritis (CL-RA) From Treatment Initiation to Month 12 By Treatment Arm

    Baseline to Month 12

  • Number of Participants Who Developed Inflammatory Arthritis (IA) From Treatment Initiation to Month 12.

    Baseline to Month 12

  • Time to Development of Clinically-Apparent Rheumatoid Arthritis (CL - RA) By Treatment Arm

    Baseline to Month 36

  • Number of Participants Who Developed Inflammatory Arthritis (IA) From Treatment Initiation to Month 36

    Baseline to Month 36

  • Number of Participant Self-Reported Painful Joints By Treatment Arm

    At Week 52 and Month 36/End of Study

  • +9 more secondary outcomes

Study Arms (2)

Hydroxychloroquine Group

EXPERIMENTAL

Subjects randomized to hydroxychloroquine (HCQ). Subjects will receive 200-400 mg of HCQ (1-2 pills), based upon ideal body weight (IBW), taken daily for 12 months.

Drug: Hydroxychloroquine

Placebo Group

PLACEBO COMPARATOR

Subjects randomized to placebo HCQ. Subjects will receive 200 - 400 mg of HCQ placebo (1-2 pills), based upon IBW, taken daily for 12 months.

Drug: HCQ Placebo

Interventions

As described. Dosing will be based upon Screening IBW.

Also known as: HCQ, Plaquenil
Hydroxychloroquine Group

As described. Dosing will be based upon Screening IBW.

Also known as: Placebo
Placebo Group

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects who meet all of the following criteria are eligible for enrollment into the study:
  • Able and willing to give written informed consent and comply with requirements of the study;
  • Age ≥18 years-old at the Screening Visit; and
  • Elevation of autoantibody anti-cyclic citrullinated peptide-3 (anti-CCP3) defined by result of anti-CCP3 ≥40 units, at Screening.

You may not qualify if:

  • Subjects who meet any of the following criteria are ineligible to participate in the study:
  • A medical history of inflammatory arthritis (IA) of any type and/or rheumatic disease and immunologic disease(s) that may be associated with IA . These diseases include but are not limited to:
  • rheumatoid arthritis (RA);
  • systemic lupus erythematosus (SLE);
  • seronegative spondyloarthropathies;
  • inflammatory bowel disease;
  • Sjögren's syndrome;
  • polymyalgia rheumatic; or
  • vasculitis.
  • A medical history of:
  • congestive heart failure or functional status of New York Heart Association (NYHA) Class III or higher at the Screening Visit;
  • cardiomyopathy or significant cardiac conduction disorders;
  • chronic liver disease;
  • psoriasis (due to potential for increased risk for flare of skin disease);
  • porphyria;
  • +25 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

University of Alabama at Birmingham School of Medicine: Division of Clinical Immunology & Rheumatology

Birmingham, Alabama, 35294, United States

Location

Cedars Sinai Medical Center: Division of Rheumatology

Los Angeles, California, 90048, United States

Location

UCLA Medical Center: Division of Rheumatology

Los Angeles, California, 90095, United States

Location

University of California San Francisco, San Francisco General Hospital

San Francisco, California, 94110, United States

Location

University of Colorado School of Medicine: Division of Rheumatology

Aurora, Colorado, 80045, United States

Location

Emory Clinic at 1365 Clifton Road: Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Brigham & Women's Hospital: Department of Medicine, Rheumatology, Immunology

Boston, Massachusetts, 02115, United States

Location

University of Massachusetts Memorial Medical Center: Rheumatology

Worcester, Massachusetts, 01605, United States

Location

University of Michigan Health System: Department of Internal Medicine, Division of Rheumatology

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic, Division of Rheumatology

Rochester, Minnesota, 55905, United States

Location

University of Nebraska Medical Center: Division of Rheumatology

Omaha, Nebraska, 68198, United States

Location

Northwell Health

Great Neck, New York, 110211, United States

Location

Oklahoma Medical Research Foundation: Arthritis and Clinical Immunology Research Program

Oklahoma City, Oklahoma, 73104, United States

Location

University of Texas Southwestern Medical Center, Division of Rheumatic Diseases

Dallas, Texas, 75390, United States

Location

Related Publications (1)

  • Deane KD, Striebich CC, Feser ML, O'Dell JR, James JA, Sparks JA, Davis JM, Graf J, McMahon MA, Solow EB, Forbess L, Tiliakos A, Schiopu E, Danila MI, Horowitz DL, Kay J, Strickland CD, Guthridge JM, Arriens C, Grossman JM, Demoruelle MK, Bemis EA, Frazer-Abel A, Fleischer CL, Fox DA, Mikuls TR, Greenleaf M, York K, Walker S, Keyes-Elstein L, Byron M, Fedler J, Goldmuntz EA, Holers VM. A phase 2 trial of hydroxychloroquine in individuals at risk for rheumatoid arthritis. Arthritis Rheumatol. 2025 Aug 29. doi: 10.1002/art.43366. Online ahead of print.

Related Links

MeSH Terms

Conditions

Arthritis, Rheumatoid

Interventions

Hydroxychloroquine

Condition Hierarchy (Ancestors)

ArthritisJoint DiseasesMusculoskeletal DiseasesRheumatic DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

ChloroquineAminoquinolinesQuinolinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Limitations and Caveats

Enrollment closed at 144 randomized participants before reaching the target of 200 due to slow participant accrual. The decision was supported by a revised power analysis suggesting sufficient power for the study with reduced numbers assuming a clinically-relevant difference in RA rates between arms (50% risk for placebo \& 25% risk for HCQ). In the 1st interim analysis after enrollment closure, the follow up \& treatment of the active participants were stopped early due to evidence of futility.

Results Point of Contact

Title
Director, Clinical Research Operations Program
Organization
DAIT/NIAID

Study Officials

  • Kevin Deane, MD, PhD

    University of Colorado School of Medicine

    STUDY CHAIR
  • Michael Holers, MD

    University of Colorado School of Medicine

    STUDY CHAIR
  • Christopher Striebich, MD, PhD

    University of Colorado School of Medicine

    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
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 10, 2015

First Posted

November 11, 2015

Study Start

April 27, 2016

Primary Completion

November 1, 2022

Study Completion

November 1, 2022

Last Updated

June 12, 2025

Results First Posted

November 21, 2023

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will share

The plan is to share data in ImmPort, a long-term archive of clinical and mechanistic data from DAIT-funded grants and contracts, upon completion of the trial.

Time Frame
After completion of the study.
Access Criteria
When posted, the IPD will be available to the public.
More information

Locations