Hydroxychloroquine for Lupus

(SMILE Trial)

Recruiting at 6 trial locations

Overseen by David R Karp, MD PhD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Milton S. Hershey Medical Center

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This project is a multicenter, randomized, placebo-controlled, double-blind clinical trial that is designed to test whether treating patients who are at risk for development of lupus with hydroxychloroquine can slow accumulation of disease features. Effects on clinical progression of symptoms, patient-reported outcomes and changes in the immune markers of response will be measured and toxicity of the treatment will be assessed. This trial is a first step in testing a prevention strategy for lupus.

Research Team

Nancy J Olsen, MD

Principal Investigator

Penn State MS Hershey Medical Center

David R Karp, MD PhD

Principal Investigator

UT Southwestern Medical Center

Eligibility Criteria

This trial is for individuals aged 15-49 with a risk of developing lupus, evidenced by certain immune system markers. They must have some but not all symptoms of lupus and cannot have been treated with immunosuppressants or antimalarials before. People with other autoimmune diseases (except thyroid conditions), significant eye disease, liver problems, psoriasis, porphyria, infections like HIV or hepatitis B/C, cancer (with exceptions), or those pregnant/breastfeeding are excluded.

Inclusion Criteria

Written informed consent (and assent when applicable) obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study

Anti-nuclear antibody (ANA) titer of 1:80, or greater, as determined by immunofluorescence assay (IFA)

I am between 15 and 49 years old.

Exclusion Criteria

I have taken or am taking antimalarial drugs like hydroxychloroquine.

I take more than 10 mg of prednisone daily or changed my dose in the last 3 months.

I cannot take hydroxychloroquine due to an existing eye condition, liver issues, psoriasis, porphyria, or an allergy.

Treatment Details

Interventions

• Hydroxychloroquine (Anti-malarial)
• Placebo Oral Capsule (Anti-malarial)

Trial Overview The study tests if Hydroxychloroquine can prevent the progression of lupus in at-risk patients compared to a placebo. It's randomized and double-blind meaning neither participants nor researchers know who gets the real medicine versus the placebo. The trial will measure symptom progression, patient-reported outcomes and changes in immune response markers.

Participant Groups

Group I: Hydroxychloroquine Active Control 1 Intervention

Hydroxychloroquine will be administered as a once daily dose of 200 or 400 mg, based on the patient's weight. Treatment will be for 96 weeks.

Group II: Placebo oral capsule Placebo Group 1 Intervention

Placebo will be administered as one or two capsules as a single daily dose, based on the patient's weight. Treatment will be for 96 weeks.

Who Is Running the Clinical Trial?

Milton S. Hershey Medical Center

Lead Sponsor

Trials

515

Recruited

2,873,000+

Dr. Robert Harbaugh

Milton S. Hershey Medical Center

Chief Medical Officer since 2024

MD from Penn State College of Medicine

Don McKenna

Milton S. Hershey Medical Center

Chief Executive Officer since 2024

Master’s in Public Administration and Bachelor of Science in Business Administration and Marketing from Long Island University

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Collaborator

Trials

508

Recruited

1,090,000+

Dr. Lindsey A. Criswell

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Director since 2021

MD, MPH, DSc

Dr. Robert Colbert

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Clinical Director since 2014

MD