Treatment of Systemic Lupus Erythematosus (SLE) With N-acetylcysteine

Summary

Systemic lupus erythematosus (SLE) is a chronic inflammatory disease which often has debilitating and potentially life-threatening consequences. The cause of SLE is unknown and current therapies lack specificity and carry significant side-effects. We previously discovered the depletion of glutathione in lymphocytes of patients with SLE and associated this metabolic change with the elevation of the mitochondrial transmembrane potential.

This study will titrate to tolerance during an initial 3 month open label period and then subjects will be randomized to one of 2 arms.

It was determined by statistical analysis that each group must have 105 subjects. All subjects will be enrolled and evaluated for tolerance of NAC between dosages of 2.4 g/day and 4.8 g/day for 3 months. After A 3-month open-label dose-titration phase, SLE subjects will be randomized into 2 groups of 105 subjects either to continue the tolerated dosage of NAC or switched to equal number of placebo capsules. There will be up to seven study visits per SLE subject, including the screening and wash out visits. Visits 2-6 will be scheduled three months apart. The study will last 13 months with the wash-out visit. Each subject will donate approximately 100 ml of blood for biomarker studies at each visit. Healthy control subjects will donate blood at the same time. They will be matched to the SLE subjects by gender, age within 10 years, and ethnicity. Their blood will be used as reference for biomarker assays.

There is a consent form required to participate in the phase II study.

Conditions

• Systemic Lupus Erythematosus

Interventions

DRUG

N-acetylcysteine

Capsules of NAC, each containing 600 mg of NAC between dosages of 2.4 g to 4.8 g daily

DRUG

Placebo

placebo (sugar) twice daily, daily dosage will match that of NAC that was tolerated between daily dosages of 2.4 g and 4.8 g during the open-label titration phase.

Sponsors & Collaborators

• Cedars-Sinai Medical Center

  collaborator OTHER

• Penn State University

  collaborator OTHER

• Columbia University

  collaborator OTHER

• Hospital for Special Surgery, New York

  collaborator OTHER

• Yale University

  collaborator OTHER

• Ohio State University

  collaborator OTHER

• Oklahoma City VA Health Care System

  collaborator FED

• St. Luke's Hospital and Health Network, Pennsylvania

  collaborator OTHER

• Emory University

  collaborator OTHER

• University of Pittsburgh

  collaborator OTHER

• University of Kentucky

  collaborator OTHER

• University of Rochester

  collaborator OTHER

• State University of New York - Upstate Medical University

  lead OTHER

Principal Investigators

• Andras Perl, M.D., Ph.D. · State University of New York - Upstate Medical University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2022-05-05

Primary Completion

2027-03-31

Completion

2028-09-30

FDA Drug

Yes

Countries

• United States

Study Locations