NCT07463521招募中3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 11
- 主要终点
- Change from Baseline at Day 43 in (Myasthenia Gravis Impairment Index) MGII ocular score (Patient-Reported Outcome (PRO) part)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •Participant has any clinically significant medical or psychiatric condition (including an alcohol or drug use disorder), recent major surgery (including thymectomy [within 3 months of Screening], solid organ, stem cell or marrow transplant), planned major surgery (including thymectomy) during study participation, and/or significant laboratory abnormality that, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
- •Participant has been diagnosed with other diseases that lead to eyelid dropping, peripheral muscle weakness, or diplopia, including other autoimmune diseases that would interfere with an accurate assessment of the oMG clinical symptoms or other neurological diseases, such as congenital myasthenic syndromes, mitochondrial diseases, and muscular dystrophies.
- •Participant has a known hypersensitivity to other anti-Fc receptor (FcRn) medications, to any components of the study medication (including the excipient polysorbate 80) or has a known history of hyperprolinemia, since L-proline is a constituent of the rozanolixizumab formulation.
- •Participant has active neoplastic disease or has received treatment for neoplastic disease within 5 years of study entry (except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the uterine cervix, carcinoma in situ of the breast, or incidental histological findings of prostate cancer [Tumor, Node, Metastasis (TNM) stage T1a or T1b] that has been definitely treated with standard of care approaches).
- •Participant has a clinically relevant active infection (eg, tuberculosis (TB) infection) or a history of serious infection (resulting in hospitalization or requiring intravenous (iv) antibiotic treatment) within 6 weeks before the Baseline Visit.
- •Participant has renal impairment, defined as glomerular filtration rate less than 30milliliter/min/1.73m2 at Screening.
- •Participant has been previously treated with FcRn inhibitors.
- •Participant has been treated with any of the immunosuppressive medications, biologics, or other therapies, in the specified prohibitive timeframe.
研究组 & 干预措施
Rozanolixizumab
Experimental
After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive rozanolixizumab. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study.
干预措施: Rozanolixizumab (Drug)
Placebo
Placebo Comparator
After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive placebo. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline at Day 43 in (Myasthenia Gravis Impairment Index) MGII ocular score (Patient-Reported Outcome (PRO) part)
时间窗: At Day 43
MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week" ie, the last 7 days.
次要结局
- Change from Baseline at Day 43 in Myasthenia Gravis Symptoms Patient-Reported Outcome (MGSPRO) ocular muscle weakness scale score(At Day 43)
- Change from Baseline at Day 43 in Myasthenia Gravis Quality of Life 15-item Scale (revised version) (MG-QoL15r) total score(At Day 43)
- Change from Baseline at Day 43 in Myasthenia Gravis Activities of Daily Living (MG-ADL) ocular subdomain score(At Day 43)
- Change from Baseline at Day 43 in Myasthenia Gravis Impairment Index (MGII) ocular score (Physical Examination part)(At Day 43)
- Incidence of treatment-emergent adverse events (TEAEs)(Up to Week 13)
- Incidence of treatment-emergent serious adverse events (TESAEs)(Up to Week 13)
- Incidence of TEAEs leading to permanent withdrawal of study treatment(Up to Week 13)
研究者
研究点 (11)
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