PF-06823859 for Myositis

Recruiting at 176 trial locations

Overseen By Pfizer CT.gov Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Pfizer

Must be taking: Corticosteroids, Immunosuppressants

Must not be taking: Anti-interferon beta

Disqualifiers: Cancer, Infections, Neurological disorders, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 48 weeks

318 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new medicine called dazukibart (also known as PF-06823859) to determine its effectiveness for muscle inflammation disorders, specifically dermatomyositis (DM) and polymyositis (PM). These conditions cause muscle weakness, making daily activities like climbing stairs or walking difficult. Participants will receive either the study medicine or a placebo through an IV every four weeks for about a year. Suitable candidates for this trial have active DM or PM and are already taking a stable dose of a corticosteroid or immunosuppressant, which are medications that reduce inflammation. The trial aims to assess the safety and effectiveness of dazukibart for treating these muscle disorders. 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 requires participants to be on a stable dose of their current corticosteroid or immunosuppressant medication. The protocol does not specify if other medications need to be stopped, but it seems you can continue your current treatment if it meets the study's criteria.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that dazukibart is generally well-tolerated by people with dermatomyositis (DM). Studies have found that this treatment significantly reduces disease activity. Patients generally responded well, suggesting it may be safe to use.

Current evidence suggests that dazukibart could be a promising option for managing symptoms in people with DM and polymyositis (PM). However, like any treatment, some risks may exist, and individual experiences can vary. Participants considering this trial should weigh the potential benefits against any possible side effects. ^{1 2 3 4 5}

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

Most treatments for myositis work by reducing inflammation or suppressing the immune system to slow down the muscle damage, often using corticosteroids or immunosuppressants. But PF-06823859 is different because it targets a specific protein involved in the immune response, potentially offering more precise control over the disease. This innovative approach could lead to fewer side effects compared to traditional therapies, which often have broad, non-specific effects on the immune system. Researchers are excited about PF-06823859 because it is administered via intravenous infusion every four weeks, which may provide a more convenient dosing schedule and improve patient adherence compared to daily medications.

What evidence suggests that PF-06823859 might be an effective treatment for myositis?

In this trial, participants will receive either PF-06823859 (dazukibart) or a placebo. Previous studies have shown that dazukibart significantly reduces symptoms in patients with dermatomyositis and polymyositis. This treatment blocks a protein called interferon beta, which can cause inflammation, thereby reducing muscle inflammation and weakness. Patients who received dazukibart experienced quick improvements and generally tolerated the treatment well. These findings suggest that dazukibart could be a promising option for easing symptoms in people with these conditions. ^{1 2 3 4 6}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

This trial is for adults over 18 with active Dermatomyositis (DM) or Polymyositis (PM), who are on a stable dose of corticosteroids or immunosuppressants. It's not suitable for those not meeting the specific disease criteria or unable to follow the study protocol.

Inclusion Criteria

I have active dermatomyositis or polymyositis.

I am on a stable dose of my regular medications.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive PF-06823859 or placebo via intravenous infusion every 4 weeks

48 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PF-06823859
• Placebo

Trial Overview

The trial tests PF-06823859, given as an IV infusion every 4 weeks against a placebo, to see if it's safe and effective in treating muscle inflammation and weakness caused by DM and PM over approximately 13 months with up to 16 visits.

How Is the Trial Designed?

Group I: PF-06823859 Experimental Treatment 1 Intervention

Participants will receive PF-06823859 via intravenous infusion every 4 weeks.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo via intravenous infusion every 4 weeks.

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. Paik, JJ., Casciola-Rosen, L., Shin, JY., et al.[2022]

In a study of 250 patients with Idiopathic Inflammatory Myositis (IIM), myositis-specific antibodies (MSA) and myositis-associated antibodies (MAA) were found in 59.2% of cases, indicating a strong association between these antibodies and distinct clinical phenotypes of IIM.

The study identified specific clinical associations for various antibodies, such as anti-Jo-1 linked to mechanic's hands and interstitial lung disease, and anti-TIF-1γ associated with a higher risk of malignancy, highlighting the importance of antibody testing in understanding and managing IIM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Myositis-specific and myositis-associated autoantibodies in a large Indian cohort of inflammatory myositis. Gupta, L., Naveen, R., Gaur, P., et al.[2021]

In a phase 2 study involving boys aged 4 to 8 with Duchenne muscular dystrophy, edasalonexent was found to be well-tolerated, with mild gastrointestinal side effects and no serious adverse events reported.

The treatment with edasalonexent at 100 mg/kg/day showed promising results in slowing disease progression and preserving muscle function, alongside reductions in NF-κB-regulated gene levels and improvements in muscle health biomarkers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial. Finkel, RS., Finanger, E., Vandenborne, K., et al.[2021]

Current treatment for Duchenne muscular dystrophy (DMD) primarily involves corticosteroids, which have limited effectiveness and significant side effects; however, targeting specific inflammatory factors in skeletal muscles shows promise for better therapeutic outcomes.

Research suggests that nonsteroidal anti-inflammatory drugs (NSAIDs) targeting key inflammatory pathways, such as calcium channels and nuclear factor-κB signaling, could be more effective and safer than steroids, especially when combined with therapies aimed at restoring dystrophin protein levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors. Miyatake, S., Shimizu-Motohashi, Y., Takeda, S., et al.[2018]

In a study of 3067 cases of idiopathic inflammatory myopathy (IIM) across 11 countries, smoking was linked to increased risks of interstitial lung disease, dysphagia, malignancy, and cardiac involvement, highlighting the significant impact of environmental factors on disease severity.

The research found that extramuscular symptoms, such as dysphagia and cardiac issues, were common in IIM patients and correlated with higher Health Assessment Questionnaire scores, indicating a greater disease burden and the need for comprehensive management strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The EuroMyositis registry: an international collaborative tool to facilitate myositis research. Lilleker, JB., Vencovsky, J., Wang, G., et al.[2021]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/39798982/

Efficacy, safety, and target engagement of dazukibart, an ...

Dazukibart resulted in a pronounced reduction in disease activity and was generally well tolerated, supporting IFNβ inhibition as a highly promising ...

2.

thelancet.com

thelancet.com/journals/lancet/article/PIIS0140-6736(24)02071-3/abstract

Efficacy, safety, and target engagement of dazukibart, an ...

Efficacy, safety, and target engagement of dazukibart, an IFNβ specific monoclonal antibody, in adults with dermatomyositis: a multicentre, double-blind, ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05895786

NCT05895786 | A Study to Understand How the ...

The purpose of the study is to understand how the study medicine PF-06823859 (dazukibart) works in people with idiopathic inflammatory myopathies (DM and ...

4.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0140673624020713

Efficacy, safety, and target engagement of dazukibart, an ...

The results showed that dazukibart had a rapid and pronounced reduction in disease activity in patients. Declaration of interests. DF received ...

5.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12272793/

Dazukibart for dermatomyositis: expanding the therapeutic ...

In conclusion, this study adds dazukibart to the expanding arsenal of drugs targeting the interferon pathway in dermatomyositis. Over the coming ...

6.

pfizer.com

pfizer.com/sites/default/files/plsr-studies/C0251002%20Plain%20Language%20Study%20Results%20Summary%20%28PLSRS%29%20Phase%202-4.pdf

Clinical Study Results

The main purpose of Stage 3 of this study was to learn about the effects, safety, and tolerability of PF-06823859 in DM participants with muscle.