A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous Administered by Prefilled Syringe in Adult Participants With Sjogren's Disease-Associated Sensorimotor or Sensory Polyneuropathy
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- argenx
- 入组人数
- 75
- 主要终点
- Change from baseline in modified Toronto Clinical Neuropathy Score (mTCNS) total score at the end of Part A
研究概览
简要总结
This study aims to assess the efficacy and safety of efgartigimod PH20 in adults with Sjogren's Disease (SjD) associated with nerve damage. The study will assess how efgartigimod PH20 affects symptoms of SjD specifically linked to the peripheral nervous system; the safety and tolerability of efgartigimod PH20; and whether receiving efgartigimod PH20 affects the participant's quality of life.
详细描述
The study will be conducted in adult participants with moderate-to-severe SjD and SjD-associated sensorimotor polyneuropathy (SMPN) or sensory polyneuropathy (SPN). The study consists of two parts. Part A is a randomized, double-blinded, placebo-controlled, parallel-group treatment period designed to evaluate the efficacy and safety of efgartigimod hyaluronidase (PH20) subcutaneously (SC) or placebo. Following the completion of part A, the double-blinded treatment period (DBTP) participants will enter part B, an open-label treatment period (OLTP) to assess long-term safety, tolerability, and durability of response of efgartigimod PH20 SC.
The overall study duration will be up to 108 weeks for each participant.
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
- •Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF
- •Meets the following SjD criteria: Fulfilled American College of Rheumatology and the European Alliance of Associations for Rheumatology classification 2016 SjD criteria before screening; Moderate-to-severe disease defined as a ESSDAI ≥ 5 or clinESSDAI ≥ 6 with PNS domain score of ≥ 5 (ie, a score of at least low activity) at screening; Anti-Ro/SS-A positive at a central laboratory at screening
- •Meets the following SjD-associated polyneuropathy criteria: Diagnosis of SjD-associated SMPN, SPN or sensory ganglionopathy, with neuropathy duration ≤ 5 years at screening and signs of active neuropathic disease development within the last 12 months; patients with co-existing SjD-associated small fiber neuropathy are eligible; No alternative diagnosis of neuropathy etiology; Total mTCNS ≥ 6 at screening; mTCNS sensory test score <level 3 at screening (does not apply to sensory ganglionopathy cohort where any severity is acceptable)
排除标准
- •Besides the indication under study, known medical conditions that would interfere with an accurate assessment of clinical symptoms of SjD-associated SMPN or SPN or gangliopathy, confound the study results, or puts the participant at undue risk.
- •Associated (also known as secondary) SjD, defined as overlap with another autoimmune rheumatic or systemic inflammatory condition (eg, rheumatoid arthritis, systemic lupus erythematosus, scleroderma, or idiopathic inflammatory myopathy).
- •Active fibromyalgia which is not adequately controlled in the judgment of the investigator, or participant is receiving fibromyalgia treatment that has not been stable treatment for at least 12 weeks before screening.
- •An alternative etiology for SMPN/SPN/sensory ganglionopathy or insufficient evidence of the diagnosis.
- •Any severe SjD manifestation or other health condition not adequately controlled at screening or baseline that may put the participant at undue risk based on the investigator's opinion.
- •Comorbidities (eg, asthma, chronic obstructive pulmonary disease) which have required 3 or more courses of systemic (oral, IV, or IM) glucocorticoids within the previous 12 months.
- •History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥ 3 years; before first IMP administration.
研究组 & 干预措施
Double-blinded treatment period (DBTP): Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 SC during the double-blinded treatment period
干预措施: efgartigimod PH20 SC (Biological)
Double-blinded treatment period (DBTP): Placebo PH20 SC
Participants receiving placebo PH20 SC during the double-blinded treatment period
干预措施: Placebo PH20 SC (Other)
Open-label treatment period (OLTP)
Participants receiving efgartigimod PH20 SC during the open-label treatment period
干预措施: efgartigimod PH20 SC (Biological)
结局指标
主要结局
Change from baseline in modified Toronto Clinical Neuropathy Score (mTCNS) total score at the end of Part A
时间窗: Up to 48 weeks
The mTCNS (modified Toronto Clinical Neuropathy Score) is a clinician-reported outcome measure derived from the original TCNS to improve sensitivity to early and mild neuropathy and to enhance feasibility and consistency inclinical research settings. The total mTCNS score is calculated as the sum of the symptom and sensory subscores, yielding a total possible score range of 0 to 33, with higher scores indicating greater neuropathy severity.
次要结局
- Change from baseline in modified Toronto Clinical Neuropathy Score (mTCNS) total score over time(up to 96 weeks)
- Change from baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score at the end of part A and over time(up to 96 weeks)
- Change from baseline in clinical EULAR Sjögren's Syndrome Disease Activity Index (clinESSDAI) score at the end of part A and over time(up to 96 weeks)
- Change from baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) score at the end of part A and over time(up to 96 weeks)
- Proportion of participants with low disease activity (clinESSDAI < 5) at the end of part A and over time(up to 96 weeks)
- Incidence of AEs(Up to 104 weeks)
- Change from baseline in Neuropathic Pain Questionnaire (NPQ) at the end of part A and over time(up to 96 weeks)
- Change from baseline in the Difficulty Thinking Numerical Rating Scale (NRS) at the end of part A and over time(up to 96 weeks)
- Change from baseline in Patient Global Impression of Severity (PGIS) scores at the end of part A and over time(up to 96 weeks)
- Change from baseline in Patient Global Impression of Change (PGIC) scores at the end of part A and over time(up to 96 weeks)
- Change from baseline in Clinical Global Impression of Severity (CGIS) scores at the end of part A and over time(up to 96 weeks)
- Change from baseline in Clinical Global Impression of Change (CGIC) scores at the end of part A and over time(up to 96 weeks)
- Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue) scores at the end of part A and over time(up to 96 weeks)