PF-06823859 for Myositis

Recruiting at 145 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

270 Participants Needed

Trial Summary

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.

What makes the drug PF-06823859 unique for treating myositis?

PF-06823859, also known as Dazukibart, is a novel treatment for myositis, a condition with limited standard treatment options. Its uniqueness may lie in its specific mechanism of action or formulation, which is not detailed in the available research, but it is being compared to a placebo in clinical trials to assess its effectiveness. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing a new medicine called PF-06823859 for adults with muscle inflammation diseases DM and PM. The goal is to see if it can safely reduce muscle inflammation and weakness.

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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 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 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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Myositis-specific and myositis-associated autoantibodies in a large Indian cohort of inflammatory myositis. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. [2022]

3. England pubmed.ncbi.nlm.nih.gov

The EuroMyositis registry: an international collaborative tool to facilitate myositis research. [2021]

4. England 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. [2021]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors. [2018]