Pozelimab and Cemdisiran Therapy for Sporadic Inclusion Body Myositis
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
Recruiting
Early Phase 1Interventional Study
Sporadic Inclusion Body Myositis (sIBM)Idiopathic Inflammatory Myopathies
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
45 – 75
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
10 (estimated)
Sponsor
Austin Neuromuscular Center · Other
Who this trial is looking for
This trial is looking for adults aged 45 to 75 who have sporadic inclusion body myositis. Participants will receive a combination therapy and must attend regular clinic visits during the study.
Are You a Good Fit for This Trial?
Rules you outActive tuberculosisHepatitis BHepatitis CHIV
You may be able to join if
I am between 45 and 75 years old.
I have been diagnosed with sporadic inclusion body myositis.
I can walk at least 20 feet with or without help from walking devices.
I understand and can fill out study-related questionnaires.
I am willing to attend all clinic visits and follow the study procedures.
I can provide signed informed consent.
If female, I am using an acceptable birth control method or am surgically sterilized.
If male, I am using a reliable method of birth control or abstinent.
You may not be able to join if
I have another neurological or musculoskeletal condition that affects my mobility.
I have an active cancer.
I have a significant illness that may affect my participation.
I have previously received treatment with a complement inhibitor.
I have a history of meningococcal disease.
I am pregnant, planning to become pregnant, or breastfeeding.
I have a recent systemic infection.
I have been involved in another clinical trial in the past 30 days.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Trial Locations
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Status
Eligibility Criteria
Inclusion Criteria:
* 1\. Age ≥45 years to 75 years of age 2. Diagnosed with sIBM based on the ENMC IBM Research Diagnostic Criteria 3. Willing and able to comply with clinic visits and study-related procedures. 4. Provide informed consent signed by the study patient or legally acceptable represent…
Inclusion Criteria:
* 1\. Age ≥45 years to 75 years of age 2. Diagnosed with sIBM based on the ENMC IBM Research Diagnostic Criteria 3. Willing and able to comply with clinic visits and study-related procedures. 4. Provide informed consent signed by the study patient or legally acceptable representative.
5\. Able to understand and complete study-related questionnaires. 6. Able to ambulate at least 20 ft/6 meters with or without assistive device Once arising from the chair, the participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or walls for support.
7\. If female, the subject must be: (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation. or (b) of childbearing potential and using a birth control method, such as:
•Combined (estrogen and progestogen-containing) hormonal. contraception associated with inhibition of ovulation:
o Oral
* Intravaginal
* Transdermal
* Progestogen-only hormonal contraception associated with inhibition of ovulation:
* Oral
* Injectable
* Implantable
• Intrauterine device
• Intrauterine hormone-releasing system
• Bilateral tubal occlusion
* Vasectomized partner
* Sexual abstinence or of non-childbearing potential (i.e., no menses for ≥12 consecutive months without any other underlying medical cause) The subject must agree to continue using her selected method of birth control with her sexual partner during the study and for 120 days after study completion.
8\. If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or maintain total abstinence from sexual intercourse. The subject must agree to continue using his selected method of birth control with his sexual partner during the study and for 120 days after the study completion.
Exclusion Criteria:
* 1\. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson's Disease) or musculoskeletal conditions (e.g., severe osteoarthritis) causing mobility impairment.
2\. Any known active malignancy 3. Significant illness or history of significant illness that, in the investigator's opinion, may adversely affect the patient's participation in this study.
4\. Any previous treatment with a complement inhibitor 5. History of meningococcal disease 6. No documented meningococcal and pneumococcal vaccination within 5 years prior to screening visit unless vaccination will be administered during the screening period and prior to initiation of study treatment.
7\. Known contraindication to meningococcal (group ACWY conjugate and group B vaccines) and pneumococcal vaccines.
8\. Subject unwilling to receive meningococcal and pneumococcal vaccinations. 9. Patients with screening serum ALT levels \>3×ULN and/or total bilirubin \>2×ULN (unless bilirubin elevation is due to suspected Gilbert's syndrome).
10\. Active or recent systemic infection within 2 weeks prior to Baseline. 11. Pregnant, planning to become pregnant, or lactating female subjects. 12. Treatment with an experimental drug within 30 days or 5 half-lives of the experimental drug.
13\. Participation in another concurrent interventional clinical trial. 14. Subjects with functional and anatomic asplenia 15. Subjects with positive tests for hepatitis B, hepatitis C, or HIV at screening 16. Presence of active or latent tuberculosis 17. Presence of any other form of myositis or myopathy or myositis overlap with other autoimmune diseases 18. Presence of other autoimmune or autoinflammatory diseases
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