What side effects are associated with this type of intervention?

Common treatments for Lupus include hydroxychloroquine, glucocorticoids, and immunosuppressive agents such as cyclophosphamide, mycophenolate, and rituximab. Hydroxychloroquine is generally well-tolerated but can cause gastrointestinal upset and, rarely, retinal toxicity. Glucocorticoids can lead to side effects like hyperglycemia, increased infection risk, bone loss, and neuropsychiatric symptoms. Cyclophosphamide may cause bladder toxicity, myelosuppression, and infertility. Mycophenolate can lead to gastrointestinal symptoms and increased infection risk. Rituximab is associated with infusion reactions, serum-sickness-like reactions, and, rarely, severe infections like progressive multifocal leukoencephalopathy.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of systemic lupus erythematosus (SLE). Notably, belimumab, a monoclonal antibody that inhibits B-lymphocyte stimulator (BLyS), was approved in 2011 as the first new treatment for lupus in over 50 years. Additionally, hydroxychloroquine, an antimalarial drug, and corticosteroids like prednisone are commonly used to manage lupus symptoms. More recently, voclosporin, a calcineurin inhibitor, was approved in 2021 for lupus nephritis, a severe kidney inflammation caused by lupus. These treatments aim to modulate the immune system, similar to the investigational approach of Enpatoran.

What other types of research exist?

Promising novel alternatives to Enpatoran for Lupus patients include hydroxychloroquine, which may help reduce proteinuria when added to angiotensin inhibition, and investigational agents such as atacicept (an immunomodulator) and complement inhibitors like avacopan, iptacopan, narsoplimab, cemdisiran, and ravulizumab. These agents are being evaluated in clinical trials for their potential benefits in treating lupus.

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Small Molecule

Enpatoran for Lupus

Phase 2

Waitlist Available

Research Sponsored by EMD Serono Research & Development Institute, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Active SLE with presence of: CLASI-A >= 8 and BILAG 2004 1B, C, D (that is [i.e.], No BILAG 2004 A and No BILAG 2004 >= 2B) or BILAG 2004 >= 1A or 2B and 1 or 2 of the following: Hybrid Safety of Estrogens in Systemic Lupus Erythematosus National Assessment (SELENA)-SLEDAI >= 6 at Screening Visit and confirmed clinical hybrid SELENA-SLEDAI >= 4 (excluding laboratory parameters) at Day 1 Visit and/or CLASI-A >= 8

Active CLE (SCLE and/or DLE) with a CLE disease area and activity index (CLASI-A) >= 8

Must not have

History of malignancy

History of uncontrolled seizures or other neurological disorder

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 up to week 24

Summary

This trial tests an oral medication called Enpatoran on patients with different types of lupus. It aims to find out if the drug is safe and effective in reducing lupus symptoms.

Who is the study for?

The WILLOW study is for people with systemic or cutaneous lupus who are on a stable dose of standard lupus treatments. Participants should have active skin symptoms scored at least 8 on the CLASI-A scale, and may also have certain levels of overall disease activity as measured by BILAG 2004 and SELENA-SLEDAI scores.

What is being tested?

This study tests different doses of Enpatoran, an oral medication, against a placebo over 24 weeks to see how well it works and how safe it is for treating lupus. The trial randomly assigns participants to receive either low, medium, high doses of Enpatoran or a placebo in a double-blind setting.

What are the potential side effects?

While specific side effects for Enpatoran aren't listed here, common side effects from drugs like this can include stomach issues, headaches, fatigue, potential liver changes and possibly increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have active lupus with specific skin and organ involvement.

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My skin condition due to lupus is active and severe.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had cancer before.

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I do not have uncontrolled seizures or other serious brain problems.

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I do not have any serious ongoing infections.

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I do not have uncontrolled heart issues, lupus affecting my kidneys, or active brain disorders.

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I have skin conditions not related to lupus.

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I have an autoimmune disease that is not lupus.

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I have or had HIV, hepatitis B, or hepatitis C.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 up to week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 up to week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 up to week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Cohort A: Clinically Meaningful Corticosteroids (CS) Reduction

Adrenal Cortex Hormones

Trial Design

8Treatment groups

Experimental Treatment

Placebo Group

Group I: Cohort B (Part 2): Enpatoran medium doseExperimental Treatment1 Intervention

Participants with active SLE who have moderate to high systemic disease activity (BILAG A/2B) with 1 or 2 of the following: CLASI-A \>= 8 and/or SLEDAI \>= 6 will be enrolled in Cohort B to receive medium dose of Enpatoran.

Group II: Cohort B (Part 2): Enpatoran low doseExperimental Treatment1 Intervention

Group III: Cohort B (Part 1 + Part 2): Enpatoran high doseExperimental Treatment1 Intervention

Group IV: Cohort A: Enpatoran medium doseExperimental Treatment1 Intervention

Participants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive medium dose of Enpatoran.

Group V: Cohort A: Enpatoran low doseExperimental Treatment1 Intervention

Participants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive low dose of Enpatoran.

Group VI: Cohort A: Enpatoran high doseExperimental Treatment1 Intervention

Participants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive high dose of Enpatoran.

Group VII: Cohort A: PlaceboPlacebo Group1 Intervention

Participants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (Cutaneous Lupus Erythematosus Disease Area and Severity Index \[CLASI-A\] greater than or equal to \[\>=\] 8) will be enrolled in Cohort A to receive placebo matched to Enpatoran.

Group VIII: Cohort B (Part 1 + Part 2): PlaceboPlacebo Group1 Intervention

Participants with active SLE who have moderate to high systemic disease activity (British Isles Lupus Assessment Group \[BILAG A/2B\]) with 1 or 2 of the following: CLASI-A \>= 8 and/or Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) \>= 6 will be enrolled in Cohort B to receive placebo matched to Enpatoran .

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for systemic lupus erythematosus (SLE) include hydroxychloroquine, glucocorticoids, and immunosuppressive agents like azathioprine and mycophenolate mofetil. Hydroxychloroquine works by modulating the immune system and reducing inflammation, which helps control lupus symptoms and prevent flares. Glucocorticoids suppress the immune response and inflammation, providing rapid relief from severe symptoms. Immunosuppressive agents inhibit the proliferation of immune cells, reducing the overall immune activity that causes tissue damage in lupus. Understanding these mechanisms is crucial for lupus patients as it helps tailor treatments to manage disease activity effectively and minimize organ damage.

New Trials in Lupus and where Are we Going.[Clinical practice guidelines for systemic lupus erythematosus: Recommendations for general clinical management].Biologicals for the treatment of systemic lupus erythematosus: current status and emerging therapies.