What side effects are associated with this type of intervention?

Common treatments for dermatomyositis, such as glucocorticoids, methotrexate, mycophenolate mofetil, and calcineurin inhibitors, can have various side effects. Glucocorticoids may cause weight gain, osteoporosis, hypertension, and increased risk of infections. Methotrexate can lead to liver toxicity, bone marrow suppression, and gastrointestinal issues. Mycophenolate mofetil is associated with gastrointestinal disturbances and increased risk of infections. Calcineurin inhibitors like cyclosporine and tacrolimus can cause nephrotoxicity, hypertension, and increased susceptibility to infections. JAK inhibitors, similar to Brepocitinib, may cause infections, blood clots, and elevated liver enzymes.

What prior FDA approvals exist?

Currently, there are no specific FDA-approved treatments for dermatomyositis that target the TYK2/JAK1 pathway, like Brepocitinib. Traditional treatments for dermatomyositis have included immunosuppressive agents such as methotrexate, azathioprine, and mycophenolate mofetil, as well as glucocorticoids. Rituximab, a biologic agent, has also been used in refractory cases. The study of Brepocitinib represents an exploration into newer targeted therapies for this condition.

What other types of research exist?

Promising novel alternatives to Brepocitinib for Dermatomyositis patients in 2024 may include other JAK inhibitors such as Filgotinib, which has shown efficacy in autoimmune conditions like psoriatic arthritis. Additionally, newer biologics targeting specific immune pathways involved in Dermatomyositis could also be considered, though specific names and trial results would need to be discussed with a healthcare provider.

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TYK2/JAK1 Inhibitor

Brepocitinib for Dermatomyositis (VALOR Trial)

Phase 3

Recruiting

Research Sponsored by Priovant Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies

Adult subjects (18-75 years old)

Must not have

Dermatomyositis with end-stage organ involvement

Dermatomyositis with irreversible muscle involvement

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

Pivotal Trial

Summary

This trial tests brepocitinib, a drug that blocks inflammation-causing proteins, in adults with dermatomyositis. The goal is to see if it improves symptoms like muscle weakness and skin rashes by reducing inflammation. Another similar drug has shown positive results in treating difficult cases of dermatomyositis.

Who is the study for?

Adults aged 18-75 with dermatomyositis, weighing between 40-130 kg and a BMI under 40. Participants must have active muscle and skin disease, may be on or have had corticosteroids or immunosuppressants, and meet specific diagnostic criteria. Excluded are those with recent cancers (with some exceptions), high thrombosis risk, severe infections, irreversible muscle damage from the disease, end-stage organ involvement, certain other diseases or conditions.

What is being tested?

The trial is testing Brepocitinib's effectiveness compared to a placebo in adults with dermatomyositis over a year. It measures improvement using the Total Improvement Score. After this phase, participants can join an open-label extension for another year where everyone receives Brepocitinib.

What are the potential side effects?

As Brepocitinib is a TYK2/JAK1 inhibitor that affects immune function, potential side effects could include increased risk of infections, changes in blood counts leading to anemia or clotting issues; liver enzyme alterations; cholesterol level changes; headache; respiratory tract infections; nausea.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with dermatomyositis.

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I am between 18 and 75 years old.

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I have ongoing muscle and skin conditions.

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My weight is between 40kg and 130kg, and my BMI is less than 40.

Exclusion Criteria

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

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My dermatomyositis has severely affected my organs.

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I have dermatomyositis with permanent muscle damage.

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I have a disorder that affects my lymph cells.

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I currently have cancer.

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I have dermatomyositis related to my cancer.

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I currently have or recently had an infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Total Improvement Score (TIS) at Week 52

Secondary study objectives

Change from baseline in HAQ Disability Index score at Week 52

Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity Score after 52 weeks of brepocitinib administration QD, in comparison to placebo

Normalized change from baseline area under the concentration-time curve (AUC) in oral prednisone-equivalent dose through Week 52

+1 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Brepocitinib Dose Level 2 PO QDExperimental Treatment1 Intervention

Group II: Brepocitinib Dose Level 1 PO QDExperimental Treatment1 Intervention

Group III: Placebo PO QDPlacebo Group1 Intervention

0 / 10Eligibility 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

Dermatomyositis (DM) treatments primarily focus on modulating the immune system to reduce inflammation and muscle damage. Common treatments include corticosteroids, immunosuppressants like methotrexate and azathioprine, and biologics such as rituximab. These therapies work by suppressing different components of the immune response. Brepocitinib, a TYK2/JAK1 inhibitor, targets the JAK-STAT pathway, which is essential for cytokine signaling in inflammation. By inhibiting this pathway, Brepocitinib and similar drugs can potentially reduce immune-mediated damage in DM, offering a targeted approach to managing the disease.

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