New drug duo aims to slow muscle decline in rare disease
NCT ID NCT06479863
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tests whether a combination of two drugs, pozelimab and cemdisiran, can improve physical function in people with sporadic inclusion body myositis (sIBM), a rare muscle disease that causes progressive weakness. Ten adults aged 45 to 75 with sIBM will receive the treatment and be monitored for changes in muscle strength and daily activities. The goal is to see if the therapy can help control the disease and ease symptoms.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
-
About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Aug 2024
- Expected to finish
-
Aug 2027
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
45 to 75 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Idiopathic inflammatory myopathies are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The study's own enquiry address
This study publishes an address for enquiries. See it below .
-
The places running it
2 sites. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Email: •••••@•••••
Locations
-
Austin Neuromuscular Center
RECRUITINGAustin, Texas, 78759, United States
-
Austin Neuromuscular Center
RECRUITINGAustin, Texas, 78759, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new pill ease muscle weakness in myositis?
- Immune clues in the blood may foretell heart trouble
- Can a simple blood test unlock the secrets of brain autoimmune diseases?
- New tool could predict who will suffer from steroid side effects
- New study aims to unlock secrets of rare muscle disease
- 5,000 patients to help unlock secrets of inflammatory heart disease