241 Participants Needed

Brepocitinib for Dermatomyositis

(VALOR Trial)

Recruiting at 116 trial locations
CT
Overseen ByClinical Trial Administrator
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Priovant Therapeutics, Inc.
Must be taking: Corticosteroids, Hydroxychloroquine, Immunosuppressants
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it mentions that participants can have prior or current therapy with corticosteroids, hydroxychloroquine, and one non-steroid immunosuppressant, so you might be able to continue these.

What data supports the effectiveness of the drug Brepocitinib for treating dermatomyositis?

Brepocitinib is a JAK inhibitor, similar to tofacitinib and peficitinib, which have shown effectiveness in treating dermatomyositis, a condition where the immune system attacks the skin and muscles. These drugs have helped patients who did not respond to other treatments, suggesting that Brepocitinib might also be effective.12345

How is the drug Brepocitinib unique for treating dermatomyositis?

Brepocitinib is unique because it is a JAK inhibitor, similar to other drugs like tofacitinib and baricitinib, which have shown effectiveness in treating refractory dermatomyositis. This class of drugs works by blocking specific enzymes (Janus kinases) involved in the inflammatory process, offering a novel approach for patients who do not respond to standard treatments.23467

Research Team

CC

Christina Crater, MD

Principal Investigator

Senior Director, Clinical Development

NI

Noriko Iikuni, MD

Principal Investigator

VP, Clinical Development

Eligibility Criteria

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.

Inclusion Criteria

I have ongoing muscle and skin conditions.
I am currently on or have been treated with steroids, hydroxychloroquine, or one non-steroid immunosuppressant.
My weight is between 40kg and 130kg, and my BMI is less than 40.
See 2 more

Exclusion Criteria

I am at risk for blood clots and heart disease.
I currently have cancer.
I have a disorder that affects my lymph cells.
See 7 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive brepocitinib or placebo for 52 weeks in a double-blind manner

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of brepocitinib treatment for an additional 52 weeks

52 weeks

Treatment Details

Interventions

  • Brepocitinib
Trial OverviewThe 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.
Participant Groups
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

Find a Clinic Near You

Who Is Running the Clinical Trial?

Priovant Therapeutics, Inc.

Lead Sponsor

Trials
4
Recruited
570+

Findings from Research

Tofacitinib 10 mg combined with methotrexate (MTX) and peficitinib 150 mg with MTX were found to be among the most effective treatments for rheumatoid arthritis (RA) patients who did not respond adequately to disease-modifying antirheumatic drugs (DMARDs), based on a meta-analysis of 9 randomized controlled trials involving 3836 patients.
Both tofacitinib and peficitinib did not show a significant increase in the risk of serious adverse events compared to other treatments, indicating they are safe options for RA management.
Comparison of the efficacy and safety of tofacitinib and peficitinib in patients with active rheumatoid arthritis: A Bayesian network meta-analysis of randomized controlled trials.Lee, YH., Song, GG.[2021]
In a 12-week open-label study involving 10 subjects with treatment-refractory dermatomyositis, tofacitinib demonstrated strong clinical efficacy, with all participants meeting the primary outcome of improved disease activity.
Half of the subjects showed moderate improvement, while the other half had minimal improvement, and significant reductions in disease severity were observed, particularly in skin symptoms, as indicated by a notable decrease in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score.
Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients.Paik, JJ., Casciola-Rosen, L., Shin, JY., et al.[2022]
Tofacitinib was found to be effective in improving skin symptoms in patients with classic dermatomyositis (DM) and amyopathic DM, with significant cutaneous improvement reported in those who continued treatment.
However, the drug showed minimal benefit in improving muscle strength across all myositis types, and over half of the patients discontinued treatment due to lack of effectiveness or death.
Tofacitinib therapy in refractory inflammatory myositis: a retrospective cohort study of 41 patients.Beckett, M., Tan, J., Bonnardeaux, E., et al.[2023]

References

Comparison of the efficacy and safety of tofacitinib and peficitinib in patients with active rheumatoid arthritis: A Bayesian network meta-analysis of randomized controlled trials. [2021]
Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. [2022]
Successful peficitinib addition on anti-MDA5 antibody-positive dermatomyositis refractory to triple therapy and glucocorticoid reduction. [2022]
Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis. [2022]
Tofacitinib therapy in refractory inflammatory myositis: a retrospective cohort study of 41 patients. [2023]
A Retrospective Review of Tofacitinib in the Treatment of Refractory Dermatomyositis. [2022]
Janus kinase inhibitor, tofacitinib, in refractory juvenile dermatomyositis: a retrospective multi-central study in China. [2023]