Double-blind, Placebo-controlled, Randomised, Multicentre, Adaptive, Two-stage Phase 2/3 Study Evaluating Safety and Efficacy of Three Dosages of NewGam in CIDP Patients
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Octapharma
- 入组人数
- 2
- 主要终点
- Adjusted INCAT disability score
研究概览
简要总结
NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.
详细描述
This is a Phase 2/3 study that will take place in 2 stages. The primary objective of Stage 1 (Phase 2 dose-finding part)is to determine and select one dosage from three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point). The selected NewGam dosage and placebo will be employed and compared in Stage 2.
The primary objective of Stage 2 (Phase 3 confirmatory part) is to demonstrate superiority of the maintenance dosage regimen selected at study Stage 1 over placebo in patients with CIDP as assessed by the percentage of responders.
The secondary objective is to evaluate the safety (measured by number of adverse events)and efficacy of NewGam administration in patients with CIDP compared to baseline.
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
- Double (Participant, 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
- •Patients diagnosed as having CIDP based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP ; patients with MADSAM or pure motor CIDP will be included provided they fulfil these criteria
- •Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
- •>=18 years of age
排除标准
- •Unifocal forms of CIDP
- •Pure sensory CIDP
- •MMN with conduction block
- •Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry
- •Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent plasma exchange (PE) during the last 3 months prior to baseline visit
- •Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit
- •Clinical evidence of peripheral neuropathy from another
- •Known diabetes mellitus
- •Other serious medical condition complicating assessment or treatment
- •Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever
- •Known IgA deficiency with antibodies to IgA
- •History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products, or any component of NewGam
- •Known blood hyperviscosity
研究组 & 干预措施
Dosage Arm 1
NewGam 10% 0.4 g/kg
干预措施: NewGam 10% (Drug)
Dosage Arm 2
NewGam 10% 1.0 g/kg
干预措施: NewGam 10% (Drug)
Dosage Arm 3
NewGam 10% 2.0 g/kg
干预措施: NewGam 10% (Drug)
Dosage Arm 4
Placebo 0.9% Saline
干预措施: Placebo (Drug)
结局指标
主要结局
Adjusted INCAT disability score
时间窗: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
次要结局
- Vital Signs(During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2))
- Grip Strength(Visit 9 & 13)
- Nerve Conduction Studies(Visti 9 & 13)
- Motor Impairment Assessment utlizing the Expanded MRC Sum Score(Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2))