A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis
The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis.
• Participant must be a minimum of 18 years of age inclusive at the time of signing the informed consent form (ICF)
• Participant has Myasthenia Gravis Foundation of America (MGFA) Class I with any ocular weakness at Screening through Baseline. The participant may have weakness in muscles of eye (ie, extraocular muscles that move the eyeball, including the medial rectus, lateral rectus, superior rectus, inferior rectus, superior oblique, and inferior oblique, orbicularis oculi muscles, and levator palpebrae superioris) but must have normal strength in all other facial, bulbar, and limb muscles.
• Study participant has been diagnosed with Ocular Myasthenia Gravis (oMG) with consistent ocular clinical features at Screening and supported by:
• Documented presence of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK), OR
• Documented absence of autoantibodies against AChR or MuSK; in this case, documented abnormal repetitive nerve stimulation (RNS) or single fiber electromyography (SFEMG) (as defined in the adjudication manual) and at least 1 of the following should be met:
‣ Documented positive ice test (Ptosis recovers with the ice test \[applied 2 minutes (min) to the ptotic lid\] with \>2mm improvement)
⁃ History of positive edrophonium chloride (Tensilon) test (or equivalent tests used to establish oMG diagnostic as per current practice)
⁃ Demonstrated objective improvement in oMG signs with acetylcholinesterase inhibitor (AChEIs), plasma exchange (PLEX), intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg) or corticosteroids (CSs)
• Participant has an Myasthenia Gravis Impairment Index (MGII) ocular score (Patient-Reported Outcome (PRO) part) ≥6 with at least 2 ocular items with a score of ≥2 at both Screening and Baseline visits.
• Participant reported ocular symptom(s) onset \<3 years before Screening or ≥3 years provided they have demonstrated response (ie, improvement in ptosis or diploplia) to treatment (IVIg, PLEX, SCIg, pyridostigmine, and/or CSs) in the past year.
• Participant has no pupillary abnormality except those resulting from prior localized eye disease or surgical intervention.
• Participant who:
‣ is currently receiving background treatment for oMG symptoms at the time of Screening and has been receiving treatment for oMG with a stable dose for at least 30 days prior to Screening, OR
⁃ is not receiving any background treatment at the time of Screening
• Participant has a body weight ≥35kg at Baseline.