Pozelimab + Cemdisiran for Inclusion Body Myositis
Recruiting in Palo Alto (17 mi)
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase < 1
Recruiting
Sponsor: Austin Neuromuscular Center
Must not be taking: Complement inhibitors
Disqualifiers: Other neurological, active malignancy, others
No Placebo Group
Trial Summary
What is the purpose of this trial?
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot have had previous treatment with a complement inhibitor or be participating in another clinical trial. It's best to discuss your current medications with the trial team.
What data supports the idea that Pozelimab + Cemdisiran for Inclusion Body Myositis is an effective treatment?
The available research does not provide specific data on the effectiveness of Pozelimab + Cemdisiran for Inclusion Body Myositis. Instead, it discusses other treatments like bimagrumab and alemtuzumab. Bimagrumab showed some increase in muscle mass but no significant improvement in walking distance. Alemtuzumab showed a slight reduction in muscle strength decline. Overall, the research suggests that while some treatments may slow progression, there is no strong evidence of significant improvement in muscle function for Inclusion Body Myositis.12345
What safety data is available for Pozelimab + Cemdisiran in treating Inclusion Body Myositis?
The provided research does not contain specific safety data for Pozelimab + Cemdisiran (also known as Poze-Cemdi) in the treatment of Inclusion Body Myositis. The studies mentioned focus on other treatments such as bimagrumab and alemtuzumab. Therefore, no existing safety data for Pozelimab + Cemdisiran can be extracted from these sources.12367
Research Team
Yessar M Hussain, M.D.
Principal Investigator
Austin Neuromuscular Center
Eligibility Criteria
This trial is for patients with sporadic Inclusion Body Myositis (sIBM), a muscle disease causing weakness and inflammation. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and not have conditions that could interfere with the study or pose additional risks.Inclusion Criteria
Willing and able to comply with clinic visits and study-related procedures
Provide informed consent signed by the study patient or legally acceptable representative
Able to understand and complete study-related questionnaires
See 5 more
Exclusion Criteria
I have a condition affecting my movement.
I haven't had meningococcal or pneumococcal vaccines in the last 5 years.
Known contraindication to meningococcal and pneumococcal vaccines
See 15 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks. Injections are initially administered by study staff, with subsequent injections potentially administered at home by the patient or caregiver after training.
104 weeks
In-person visit for initial injection, subsequent visits as needed for monitoring
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Treatment Details
Interventions
- Pozelimab/Cemdisiran (Monoclonal Antibodies)
Trial OverviewThe trial is testing the effectiveness of combining two drugs, Pozelimab and Cemdisiran, in treating sIBM. The goal is to see if this combination therapy can improve symptoms or halt progression of the disease.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: INJECTIONExperimental Treatment1 Intervention
patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks. SC injections will be administered by the study staff and the patient will be monitored for 30 minutes after the initial first injection. During the remainder of the study, injections can be administered at home by the patient or caregiver after injection training provided by the study staff. Designated persons will be observed to confirm their ability to perform the injections.
The dosing window of the study treatment is within ±7 days from the scheduled dose date.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Austin Neuromuscular CenterAustin, TX
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Who Is Running the Clinical Trial?
Austin Neuromuscular Center
Lead Sponsor
Trials
2
Patients Recruited
20+
Regeneron Pharmaceuticals
Industry Sponsor
Trials
690
Patients Recruited
948,000+
Founded
1988
Headquarters
Tarrytown, USA
Known For
Precision medicine
Top Products
Dupixent, EYLEA, Libtayo, Praluent
References
Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. [2020]Inclusion body myositis is an idiopathic inflammatory myopathy and the most common myopathy affecting people older than 50 years. To date, there are no effective drug treatments. We aimed to assess the safety, efficacy, and tolerability of bimagrumab-a fully human monoclonal antibody-in individuals with inclusion body myositis.
Long-term safety and tolerability of bimagrumab (BYM338) in sporadic inclusion body myositis. [2020]To assess the long-term safety and tolerability and to monitor benefits of extended use of bimagrumab in individuals with sporadic inclusion body myositis (sIBM) who completed a single-dose core study.
Effect of Alemtuzumab (CAMPATH 1-H) in patients with inclusion-body myositis. [2022]Sporadic inclusion-body myositis (sIBM) is the most common disabling, adult-onset, inflammatory myopathy histologically characterized by intense inflammation and vacuolar degeneration. In spite of T cell-mediated cytotoxicity and persistent, clonally expanded and antigen-driven endomysial T cells, the disease is resistant to immunotherapies. Alemtuzumab is a humanized monoclonal antibody that causes an immediate depletion or severe reduction of peripheral blood lymphocytes, lasting at least 6 months. We designed a proof-of-principle study to examine if one series of Alemtuzumab infusions in sIBM patients depletes not only peripheral blood lymphocytes but also endomysial T cells and alters the natural course of the disease. Thirteen sIBM patients with established 12-month natural history data received 0.3 mg/kg/day Alemtuzumab for 4 days. The study was powered to capture > or =10% increase strength 6 months after treatment. The primary end-point was disease stabilization compared to natural history, assessed by bi-monthly Quantitative Muscle Strength Testing and Medical Research Council strength measurements. Lymphocytes and T cell subsets were monitored concurrently in the blood and the repeated muscle biopsies. Alterations in the mRNA expression of inflammatory, stressor and degeneration-associated molecules were examined in the repeated biopsies. During a 12-month observation period, the patients' total strength had declined by a mean of 14.9% based on Quantitative Muscle Strength Testing. Six months after therapy, the overall decline was only 1.9% (P
Inclusion body myositis: analysis of 32 cases. [2013]Inclusion body myositis is characterized by an insidious onset, progressive indolent course, and is generally felt to be refractory to standard therapy for myositis. We reviewed the charts of 32 patients with muscle biopsy findings suggestive of inclusion body myositis. The average time from symptom onset to diagnosis was 37 months, but initially 40% were incorrectly diagnosed. Twenty-eight patients (88%) were classified as definite or probable inclusion body myositis and were treated with various combinations of prednisone and immunosuppressive agents. Sixty-eight percent of those treated experienced a decrement in function and muscle strength. Three patients exhibited longterm improvement while 12 patients experienced delayed progression, defined by short term improvement in strength or a stable functional class, All of these patients received therapy, 5 in the form of methotrexate and prednisone. All untreated patients deteriorated clinically. In summary, (1) inclusion body myositis is a clinically distinct entity which is frequently misdiagnosed initially. (2) While clinical improvement with therapy is rare, our observations support recent reports that therapy may be associated with a slower rate of clinical progression. (3) Optimal therapy remains uncertain, but the use of low dose methotrexate and prednisone may warrant further study.
[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).
Immunophenotyping of Inclusion Body Myositis Blood T and NK Cells. [2023]Label="BACKGROUND AND OBJECTIVES">To evaluate the therapeutic potential of targeting highly differentiated T cells in patients with inclusion body myositis (IBM) by establishing high-resolution mapping of killer cell lectin-like receptor subfamily G member 1 (KLRG1+) within the T and natural killer (NK) cell compartments.
Molecular treatment effects of alemtuzumab in skeletal muscles of patients with IBM. [2018]Mechanisms of inflammation and protein accumulation are crucial in inclusion body myositis (IBM). Recent evidence demonstrated that intravenous immunoglobulin failed to suppress cell-stress mediators in IBM. Here we studied the molecular changes in skeletal muscle biopsies from patients with IBM before and after treatment with alemtuzumab.
[Inclusion body myositis--a rarely recognized disorder]. [2013]Inclusion body myositis is the most common disabling inflammatory myopathy in the elderly. It is more frequent in men and after the age of 50 years. Inflammatory and degenerative features coexist. There is a T-cell mediated autoimmunity driven by in situ clonally expanded cytotoxic CD8-positive T-cells invading non-necrotic muscle fibres expressing MHC-I antigen. The hallmarks of degeneration are the deposition of protein aggregates and the formation of vesicles. The course of the disease is slow and the diagnosis is usually set after several years. The muscle weakness and wasting is assymetric, affecting predominantly distal muscles of the upper extremity and proximal muscles of the legs. The signs and clinical course can be characteristic, but the diagnosis is established by muscle biopsy. There is currently no evidence based effective treatment for sIBM. Prednisone, azathioprine, methotrexate, cyclosporine and IFN-beta failed. Oxandrolon did not improve symptoms. Treatment with intravenous immunglobuline (IVIG) induced in some patients a transient improvement of swallowing and of muscle strenght, but the overall study results were negative. A T-cell depleting monoclonal antibody (alemtuzumab), in a small uncontrolled study slowed down disease progression for a six-month period. Repeated muscle biopsies showed the reduction of T-cells in the muscle and the suppression of some degeneration associated molecules. An effective therapeutic mean should act on both aspects of the pathomechanism, on the inflammatory and the degenerative processes as well.
Inclusion body myositis: update. [2014]To examine new developments in sporadic inclusion body myositis (IBM), including updated clinical and prognostic factors, novel autoantibody associations, unique histopathologic findings, proposed new clinical diagnostic criteria, and novel therapeutic agents.