PF-06823859 for Myositis
Palo Alto (17 mi)Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Pfizer
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
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.
Is the drug PF-06823859 a promising treatment for Myositis?Based on the information provided, there is no direct evidence or research articles discussing the effectiveness of PF-06823859 for Myositis. Therefore, we cannot determine if it is a promising treatment.3491011
What safety data is available for the treatment PF-06823859 (Dazukibart) in myositis?The provided research does not contain specific safety data for PF-06823859 (Dazukibart) in the treatment of myositis. The articles focus on the classification, diagnosis, and management of idiopathic inflammatory myopathies, the role of physical therapy, and the effects of other drugs like statins and anti-inflammatory drugs in related conditions. For specific safety data on PF-06823859, further targeted research or clinical trial results would be needed.12346
Do I have to stop taking my current medications for the trial?The trial requires participants to be on a stable dose of their current corticosteroid and/or immunosuppressant medications. The protocol does not specify stopping other medications, but you should discuss your specific situation with the study team.
What data supports the idea that the drug PF-06823859 for Myositis is an effective treatment?The available research does not provide specific data on the effectiveness of PF-06823859 for Myositis. The studies mentioned focus on other treatments or aspects of Myositis, such as tofacitinib and BYM338, but do not include results for PF-06823859. Therefore, there is no direct evidence from the provided information to support the effectiveness of PF-06823859 for Myositis.578912
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 am 18 years old or older.
I have active dermatomyositis or polymyositis.
I am on a stable dose of my regular medications.
Treatment Details
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.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: PF-06823859Experimental Treatment1 Intervention
Participants will receive PF-06823859 via intravenous infusion every 4 weeks.
Group II: PlaceboPlacebo Group1 Intervention
Participants will receive placebo via intravenous infusion every 4 weeks.
Find a clinic near you
Research locations nearbySelect from list below to view details:
AARA Clinical Research - Arizona Arthritis & Rheumatology Associates- GlendaleGlendale, AZ
Mayo Clinic HospitalPhoenix, AZ
Mayo Clinic in Arizona - ScottsdaleScottsdale, AZ
200 UCLA Medical PlazaLos Angeles, CA
More Trial Locations
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Who is running the clinical trial?
PfizerLead Sponsor
References
Statin and statin-fibrate use was significantly associated with increased myositis risk in a managed care population. [2019]We quantified the risk of myositis associated with statin and fibrate drug use with other covariates within a managed care organization (MCO) population.
Classification, diagnosis, and management of idiopathic inflammatory myopathies. [2022]The detection and characterization of a large array of autoantibodies, including at least 8 different antisynthetase, anti-SRP, -200/100 (HMGCR), -Mi-2, -CADM-140 (MDA5), -SAE, -p155, -MJ (NXP-2), and -PMS1, frequently associated with distinct and well-defined clinicopathological features, allowed for significant improvement in the definition and diagnosis of idiopathic inflammatory myopathies (IIM). Classification remains difficult, with lingering divergence between the different specialties involved in IIM care, but several categories clearly stand out, including dermatomyositis (DM), overlap myositis (OM), polymyositis, necrotizing myositis, and sporadic inclusion body myositis (s-IBM). Biopsy and histological analysis remain crucial, particularly in the absence of autoantibodies, to accurately specify the diagnosis and rule out mimics such as muscular dystrophies and metabolic myopathies. Numerous infectious agents (in particular human immunodeficiency virus and human T cell lymphotrophic virus-1) and drugs (statins, tumor necrosis factor inhibitors, and proton pump inhibitors) can cause mimic IIM that must also be excluded. Pharmacological treatment, in addition to glucocorticoids and immunoglobulins, now includes mycophenolate mofetil and rituximab, which proved helpful in resistant cases, particularly rituximab in DM and OM. Exercise, initially seen as potentially deleterious, recently was shown to be efficacious and safe. IIM can thus be reasonably well controlled in most cases, although aggressive disease remains refractory to treatment, including some cases of necrotizing myopathy. Sporadic IBM still seems resistant to all medications tested to date.
Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors. [2018]Duchenne muscular dystrophy (DMD), an incurable and a progressive muscle wasting disease, is caused by the absence of dystrophin protein, leading to recurrent muscle fiber damage during contraction. The inflammatory response to fiber damage is a compelling candidate mechanism for disease exacerbation. The only established pharmacological treatment for DMD is corticosteroids to suppress muscle inflammation, however this treatment is limited by its insufficient therapeutic efficacy and considerable side effects. Recent reports show the therapeutic potential of inhibiting or enhancing pro- or anti-inflammatory factors released from DMD skeletal muscles, resulting in significant recovery from muscle atrophy and dysfunction. We discuss and review the recent findings of DMD inflammation and opportunities for drug development targeting specific releasing factors from skeletal muscles. It has been speculated that nonsteroidal anti-inflammatory drugs targeting specific inflammatory factors are more effective and have less side effects for DMD compared with steroidal drugs. For example, calcium channels, reactive oxygen species, and nuclear factor-κB signaling factors are the most promising targets as master regulators of inflammatory response in DMD skeletal muscles. If they are combined with an oligonucleotide-based exon skipping therapy to restore dystrophin expression, the anti-inflammatory drug therapies may address the present therapeutic limitation of low efficiency for DMD.
The EuroMyositis registry: an international collaborative tool to facilitate myositis research. [2021]The EuroMyositis Registry facilitates collaboration across the idiopathic inflammatory myopathy (IIM) research community. This inaugural report examines pooled Registry data.
Current Treatment for Myositis. [2023]The purpose of this review was to give an update on treatment modalities for patients with idiopathic inflammatory myopathies, or shortly myositis, excluding the subgroup inclusion body myositis, based on a literature survey on therapies used in myositis. Few controlled trials have been performed in patients with myositis; therefore, we also included a summary of open-label trials, case series, and case reports.
Physical therapy in adult inflammatory myopathy patients: a systematic review. [2020]The safety and effect of physical therapy in adult patients with idiopathic inflammatory myopathies (IIMs) are currently unclear. Considering the muscle weakness resulting from disease activity as well as from the administered drugs, these patients could benefit from an evidence-based physical therapy program. To perform a systematic review to assess safety and effects of physical therapy on the functional outcome of patients with idiopathic inflammatory myopathies in both active and quiescent disease: Pubmed, Embase, and Cochrane. Patients with one of the following idiopathic inflammatory myopathies: polymyositis, dermatomyositis, immune-mediated necrotizing myopathy, and/or overlap myositis. The intervention included several types of rehabilitation programs, from strength and resistance training to endurance training, with a minimal duration of 1 month. Studies reporting intervention-related adverse events, disease activity, and functional outcomes were eligible. The risk of bias was assessed using the Cochrane guidelines. We included five randomized controlled and seven open-label non-randomized non-controlled trials. Data on statistical significance were extracted for all the trials. Included trials were of medium-quality evidence given the low number of patients and some risk of bias factors. Physical therapy does not have a negative effect on the disease activity of idiopathic inflammatory myopathies in quiescent disease and could improve functional outcome. The physical therapy program should minimally include endurance training. A combination with resistance training might be beneficial.
[Late phase II/III study of BYM338 in patients with sporadic inclusion body myositis (RESILIENT): Japanese cohort data]. [2022]A global, randomized, double-blind placebo-controlled study was conducted to confirm that BYM338 (bimagrumab), an anti-activin type II receptor antibody, improves motor function in patients with sporadic inclusion body myositis after 52 weeks' treatment consisting of intravenous administration every 4 weeks at doses of 10, 3, and 1 mg/kg. In a Japanese sub-population (20 patients in total, 5 per dose group), no significant differences in the change from baseline of the 6-minute walking distance at Week 52 (primary endpoint) were observed between the placebo group and each BYM338 dose group. Furthermore, the lean body mass as an indicator of skeletal muscle mass increased in all BYM338 groups compared with the placebo group and the effects were dose-dependent. Overall, the Japanese sub-population showed similar trends as observed in the entire population (251 patients in total).
Promising and Upcoming Treatments in Myositis. [2023]To highlight new and emerging treatment targets in myositis.
Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. [2022]This open-label 12-week study was conducted to evaluate the efficacy and safety of tofacitinib, a JAK inhibitor, in treatment-refractory active dermatomyositis (DM).
Myositis-specific and myositis-associated autoantibodies in a large Indian cohort of inflammatory myositis. [2021]The Idiopathic Inflammatory Myositis (IIM) are heterogenous with distinct clinical phenotypes associated with specific myositis specific antibodies (MSA) and myositis associated antibodies (MAA).
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]Chronic activation of NF-κB is a key driver of muscle degeneration and suppression of muscle regeneration in Duchenne muscular dystrophy. Edasalonexent (CAT-1004) is an orally-administered novel small molecule that covalently links two bioactive compounds (salicylic acid and docosahexaenoic acid) that inhibit NF-κB. This placebo-controlled, proof-of-concept phase 2 study with open-label extension in boys ≥4-<8 years old with any dystrophin mutation examined the effect of edasalonexent (67 or 100 mg/kg/day) compared to placebo or off-treatment control. Endpoints were safety/tolerability, change from baseline in MRI T2 relaxation time of lower leg muscles and functional assessment, as well as pharmacodynamics and biomarkers. Treatment was well-tolerated and the majority of adverse events were mild, and most commonly of the gastrointestinal system (primarily diarrhea). There were no serious adverse events in the edasalonexent groups. Edasalonexent 100 mg/kg was associated with slowing of disease progression and preservation of muscle function compared to an off-treatment control period, with decrease in levels of NF-κB-regulated genes and improvements in biomarkers of muscle health and inflammation. These results support investigating edasalonexent in future trials and have informed the design of the edasalonexent phase 3 clinical trial in boys with Duchenne.
Age distribution and prevalence in different age groups of four myositis-specific autoantibodies, including anti-ARS, anti-MDA5, anti-Mi-2, and anti-TIF1γ antibodies. [2023]We accumulated the demographic information and analyzed the prevalence of myositis-specific antibodies (MSAs) in a large cohort across Japan as standard testing for MSAs becomes more widely available. This retrospective, observational, cohort study analyzed the records of individuals aged 0-99 years who are tested for serum MSAs at SRL Incorporation from January 2014 to April 2020 across Japan. An enzyme-linked immunosorbent assay testing was applied to determine the presence of anti-aminoacyl tRNA synthetase (anti-ARS), anti-Mi-2, anti-melanoma differentiation-associated gene 5 (anti-MDA5), or anti-transcriptional intermediary factor 1-γ (anti-TIF1γ) (Medical and Biological Laboratories). Anti-TIF1γ antibody was detected more in male patients than female patients. In contrast, women were predominant in patients with other MSAs. More than half of the anti-ARS or anti-TIF1γ antibody-positive patients were over 60 years old, although anti-MDA5 or anti-Mi-2-positive patients were mostly under 3-year evaluation of MSA detection in a routine diagnostic setting. This paper provides clinical images concerning the relationship between four MSA types and the distribution of sex and age in a large population.