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Also known as Pozelimab
Pozelimab/Cemdisiran · 1 trial · 2 indications
The IBM-FRS is a 10-item interviewer-administered patient-reported outcomes (PRO) measure that assesses physical function in patients with IBM. The IBMFRS includes items assessing lower body physical function, general physical function, the ability to perform certain activities of daily living, fine motor skills, and swallowing. On a Likert scale ranging from 0 (unable to perform activity) to 4 (normal), patients are asked to rate their difficulty in performing tasks relative to each concept before disease onset. Scores on the IBMFRS range from 0 to 40, with higher scores indicating better functioning.
The sIFA is an 11-item self-administered PRO measure assessing physical function in patients with sIBM. This questionnaire will be completed by the patient on a numeric rating scale ranging from 0 (no difficulty) to 10 (unable to do), with a total score ranging from 0 to 100, where 100 indicates severe limitations. P atients will be asked to rate their difficulty with performing tasks involving lower and upper body physical function, general physical function, fine motor skills, and swallowing in the past 7 days.
| Arm | Type | Description |
|---|---|---|
| INJECTION | OTHER | 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. |
| Name | Type | Description |
|---|---|---|
| Pozelimab/Cemdisiran | COMBINATION_PRODUCT | Patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks |
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...
Pozelimab is an investigational antibody being studied for CD55-deficient protein-losing enteropathy (CHAPLE disease), age-related macular degeneration, geographic atrophy, sporadic inclusion body myositis, and paroxysmal nocturnal hemoglobinuria. It is also being evaluated in combination with cemdisiran for generalized myasthenia gravis and geographic atrophy. Pozelimab is not approved and remains in clinical development.
Pozelimab is a monoclonal antibody that targets complement component C5, part of the immune system's complement cascade. By binding to C5, it is designed to block the formation of the membrane attack complex and reduce complement-mediated tissue damage. This mechanism is relevant across the diseases being studied, including CHAPLE disease and geographic atrophy.
Pozelimab is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. Regeneron is conducting clinical trials of Pozelimab both as a monotherapy and in combination with cemdisiran across multiple indications.
Pozelimab is in Phase 3 clinical development. It has completed a Phase 2 study in CD55-deficient protein-losing enteropathy and a Phase 1 study in healthy volunteers. Two Phase 3 trials are ongoing: one in generalized myasthenia gravis and one in geographic atrophy.
Pozelimab has four registered clinical trials. NCT04209634 is a completed Phase 2 study in CHAPLE disease. NCT04940364 is a completed Phase 1 study in healthy Japanese volunteers. NCT05070858 is an active Phase 3 trial in generalized myasthenia gravis. NCT06541704 is a recruiting Phase 3 trial in geographic atrophy.
Pozelimab is a single agent, while Pozelimab/Cemdisiran is a combination therapy that includes both Pozelimab and cemdisiran. The combination is being studied in clinical trials for generalized myasthenia gravis and geographic atrophy. Pozelimab alone has been studied in CHAPLE disease and healthy volunteers.