Brepocitinib for Dermatomyositis

(VALOR Trial)

Not currently recruiting at 134 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called brepocitinib for adults with dermatomyositis, a condition that causes muscle weakness and skin rashes. Researchers aim to determine if brepocitinib improves symptoms compared to a placebo (a pill with no active medicine). Participants will receive one of two doses of brepocitinib or a placebo for 52 weeks, with the option to continue with the actual medicine afterward. Ideal participants have active symptoms despite past or current treatments like steroids and no severe organ damage from dermatomyositis. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

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.

Is there any evidence suggesting that brepocitinib is likely to be safe for humans?

Research has shown that brepocitinib, a type of medication, was tested for safety in earlier studies. In these studies, brepocitinib was generally well-tolerated, with most participants not experiencing serious problems. Reported side effects were usually mild to moderate, such as headaches, nausea, and some skin reactions, while serious side effects were rare.

Brepocitinib is now in a phase 3 trial, indicating that extensive safety information exists from earlier trials. This phase focuses more on the treatment's effectiveness, providing a solid understanding of its safety. Brepocitinib is also being tested for other conditions, offering additional safety data. Always consult a healthcare provider to understand what these findings mean for individual circumstances.12345

Why do researchers think this study treatment might be promising for dermatomyositis?

Brepocitinib is unique because it targets the condition dermatomyositis with a fresh approach. Most treatments for dermatomyositis focus on suppressing the immune system broadly, but Brepocitinib works differently. It specifically inhibits Janus kinase (JAK) and tyrosine kinase 2 (TYK2), which are enzymes involved in the immune response. This targeted action could mean fewer side effects and more effective control of symptoms compared to traditional treatments. Researchers are excited about Brepocitinib because it has the potential to offer a more precise and effective treatment option for people with this challenging condition.

What evidence suggests that brepocitinib might be an effective treatment for dermatomyositis?

Research has shown that brepocitinib yields promising results for conditions like dermatomyositis, psoriasis, and psoriatic arthritis. One study found that participants taking 30 mg of brepocitinib daily experienced significantly better outcomes than those on a placebo. This trial will test different doses of brepocitinib, with some participants receiving Brepocitinib Dose Level 1 or Dose Level 2, while others receive a placebo. The drug blocks certain enzymes, TYK2 and JAK1, which play a role in inflammation. Although more research is needed specifically for dermatomyositis, these findings are encouraging.678910

Who Is on the Research Team?

CC

Christina Crater, MD

Principal Investigator

Senior Director, Clinical Development

NI

Noriko Iikuni, MD

Principal Investigator

VP, Clinical Development

Are You a Good Fit for This Trial?

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 1 more

Exclusion Criteria

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

  • Brepocitinib
Trial Overview 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.
How Is the Trial Designed?
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+

Published Research Related to This Trial

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 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]
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]

Citations

Roivant and Priovant Announce Positive Phase 3 VALOR ...Once-daily oral brepocitinib 30 mg demonstrated clinically meaningful and statistically significant improvement compared to placebo on the ...
Study Details | NCT06433999 | A 12-Week Open-Label ...A 12-Week Open-Label Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Skin-Predominant Dermatomyositis · Study Overview · Contacts and ...
Priovant mulls broad autoimmune indications for brepocitinibZimmer said brepocitinib showed efficacy in seven different indications including psoriasis, psoriatic arthritis, ulcerative colitis, and ...
Roivant and Pfizer Unveil Priovant Therapeutics ...VALOR , a single Phase 3 study evaluating brepocitinib in dermatomyositis, is currently enrolling subjects. A large Phase 2b study in systemic ...
Brepocitinib Tosilate – Application in Therapy and Current ...This article explores a Phase 3 clinical trial investigating the efficacy and safety of Brepocitinib Tosilate, a new drug for treating dermatomyositis in ...
Brepocitinib (PF-06700841) Clinical Study Report SySafety: The safety data were summarized descriptively through appropriate data tabulations, ... Safety Results: AEs, SAEs and SIEs: All AEs ...
A Study to Investigate the Efficacy and Safety ...A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
8.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/39008325/
Brepocitinib, a potent and selective TYK2/JAK1 inhibitorCurrently, the safety, tolerability, and efficacy of brepocitinib is being evaluated in the largest (n=225) double-blind placebo-controlled ...
PF-06700841 Study intervention Nameactivity on efficacy and safety outcomes. It is desirable to have ... Safety data will be presented in tabular and/or graphical format and.
Brepocitinib - Drug Targets, Indications, PatentsRoivant's TYK2/JAK1 dual inhibitor brepocitinib hit its primary and all nine key secondary endpoints in patients with dermatomyositis in the Phase III VALOR ...
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