Pozelimab and cemdisiran combination for sporadic inclusion body myositis
Tests pozelimab with cemdisiran in people with sporadic inclusion body myositis.
Key facts
- Who runs it
- Austin Neuromuscular Center
- Where
- Austin, United StatesSee the site
- Trial code
- Registry title
- Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
- Status
- Recruiting
- Phase
- Early phase 1
- People taking part
- 10 people, planned
- Started
- 8 Aug 2024
- Expected to end
- 30 Aug 2027
- Type of study
- Interventional
- Treatments tested
- Pozelimab/Cemdisiran
- Record updated
- 16 January 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults aged 45 to 75 with sporadic inclusion body myositis (sIBM), a muscle disease. It tests Pozelimab and Cemdisiran injections given together.
- Age
- 45 to 75 years
- Sex
- All sexes can join.
- Disease stage
- sIBM diagnosed with the ENMC IBM Research Diagnostic Criteria. You must be able to walk at least 20 ft/6 meters, with or without a walking aid, but without help from another person.
- Earlier treatment
- People who have ever had a complement inhibitor cannot join. No experimental drug within 30 days or 5 half-lives. Meningococcal and pneumococcal vaccines needed within 5 years, or given during screening.
Main reasons for not taking part
- Other muscle disease, myositis, or other autoimmune disease, or another condition that limits walking
- Active cancer, or past meningococcal disease
- Positive test for hepatitis B, hepatitis C or HIV, or active or latent tuberculosis
- No working spleen, or high liver test results (ALT >3×ULN or total bilirubin >2×ULN)
- Pregnant, planning pregnancy, or breastfeeding
The registry criteria, word for word
Show all 2 criteriaShow less
Who can take part
- 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; 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; 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; 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
Who cannot take part
- 1. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson's Disease) or musculoskeletal conditions (e.g., severe osteoarthritis) causing mobility impairment; 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; 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; Known contraindication to meningococcal (group ACWY conjugate and group B vaccines) and pneumococcal vaccines; 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); 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; 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
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 45 and 75 years old?
Where in United States
- Austin Neuromuscular CenterAustin, United StatesContact: Emil Hussain, ManagerTaking people
About the trial
What the trial wants to find out
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
The group in the trial
- INJECTIONOther group
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.
Treatment: Pozelimab/Cemdisiran
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
- Plain-language title
- Pozelimab and cemdisiran combination for sporadic inclusion body myositisGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 14 November 2026.
- Fit questions
- Built from NCT06479863, who can take part
- Record updated
- 16 January 2026