NCT04281472已完成2 期
A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 322
- 试验地点
- 369
- 主要终点
- Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline
研究概览
简要总结
This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Ability to understand the requirements of the trial, provide written informed consent (include consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits)
- •Male or female patient aged 18 years or older, at the time of signing the informed consent.
- •Diagnosed with probable or definite CIDP according to criteria of the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS 2010), progressing or relapsing forms.
- •CIDP Disease Activity Status (CDAS) score ≥2 at screening.
- •INCAT score ≥2 at the first run-in visit (for patients entering run-in) or stage A baseline (for treatment-naïve patients with documented evidence for worsening on the total adjusted INCAT disability score within 3 months prior to screening). Patients with an INCAT score of 2 at trial entry must have this score exclusively from the leg disability score; for patients with an INCAT score of ≥3 at trial entry, there are no specific requirements for arm or leg scores.
- •Fulfilling any of the following treatment conditions:
- •Currently treated with pulsed corticosteroids, oral corticosteroids equivalent to prednisolone/prednisone ≤10mg/day, and/or IVIg or SCIg, if this treatment has been started within the last 5 years before screening, and the patient is willing to discontinue this treatment at the first run-in visit; or
- •Without previous treatment (treatment-naive); or
- •Treatment with corticosteroids and/or IVIg or SCIg discontinued at least 6 months prior to screening Note: Patients not treated with monthly or daily corticosteroids, IVIg or SCIg for at least 6 months prior to screening are considered as equal to treatment-naïve patients.
- •Women of childbearing potential who have a negative pregnancy test at screening and a negative urine pregnancy test up to Stage A baseline.
- •Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last dose of IMP
排除标准
- •Pure sensory atypical CIDP (EFNS/PNS definition).
- •Polyneuropathy of other causes, including the following: Multifocal motor neuropathy; Monoclonal gammopathy of uncertain significance with anti-myelin associated, glycoprotein immunoglobulin M (IgM) antibodies; Hereditary demyelinating neuropathy; Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes; Lumbosacral radiculoplexus neuropathy; Polyneuropathy most likely due to diabetes mellitus; Polyneuropathy most likely due to systemic illnesses; Drug- or toxin-induced polyneuropathy.
- •Any other disease that could better explain the patient's signs and symptoms.
- •Any history of myelopathy or evidence of central demyelination.
- •Current or past history (within 12 months of screening) of alcohol, drug or medication abuse.
- •Severe psychiatric disorder (such as severe depression, psychosis, bipolar disorder), history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the patient or could affect adherence with the trial protocol.
- •Patients with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection; Active Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count ≤200 cells/mm
- •Total IgG level <6 g/L at screening.
- •Treatment with the following: Within 3 months (or 5 half-lives of the drug, whichever is longer) before screening: plasma exchange or immunoadsorption, any concomitant Fc-containing therapeutic agents or other biological, or any other investigational product; Within 6 months before screening: rituximab, alemtuzumab, any other monoclonal antibody, cyclophosphamide, interferon, tumor necrosis factor-alpha inhibitors, fingolimod, methotrexate, azathioprine, mycophenolate, any other immunomodulating or immunosuppressive medications, and oral daily corticosteroids >10 mg/day. Note: Patients using IVIg, SCIg, pulsed corticosteroids, and oral daily corticosteroids ≤10 mg/day can be included.
- •Patients who (intend to) use prohibited medications and therapies (see protocol) during the trial.
- •Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after last IMP administration.
- •Patients with any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of CIDP.
- •Patients who received a live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, sub-unit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.
- •Patients who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first IMP administration. Patients with the following cancer can be included anytime: Adequately treated basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, or Incidental histological finding of Prostate cancer (TNM [tumor, nodes, and metastases classification] stage T1a or T1b).
- •Patients who previously participated in a trial with efgartigimod and have received at least one administration of IMP.
- •Patients with known medical history of hypersensitivity to any of the ingredients of IMP.
- •Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or any other reason which could confound the results of the trial or put the patient at undue risk.
结局指标
主要结局
Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline
时间窗: Up to 48 weeks during the randomized placebo-controlled stage B
Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI)
时间窗: Up to 12 weeks during the open-label stage A
次要结局
- Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score(Up to 12 weeks during the open-label stage A)
- Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Percent Changes of Serum IgG Levels Over Time(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Changes From Stage B Baseline to Last Assessment in Stage B, in EQ-5D-5L Visual Analog Scale (VAS) Over Time(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)(Up to 12 weeks during the open-label stage A)
- Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores(Up to 12 weeks during the open-label stage A)
- Stage B: Time to CIDP Disease Progression(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in TUG Score(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events(Up to 12 weeks during the open-label stage A)
- Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20(Up to 12 weeks during the open-label stage A)
- Stage A: Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time(Up to 12 weeks during the open-label stage A)
- Stage B: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score(Up to 12 weeks during the open-label stage A)
- Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength(Up to 12 weeks during the open-label stage A)
- Stage B: Number of Participants With Improved Functional Level Compared to Stage B Baseline(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Adjusted INCAT Score(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in MRC Sum Score(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score(Up to 12 weeks during the open-label stage A)
- Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time(Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI)))
- Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time(Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI)))
- Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in 24-item I-RODS Disability Score(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Mean Grip Strength(Up to 48 weeks during the randomized placebo-controlled stage B)
- Stage B: Time to 10% Decrease in the 24-item I-RODS(Up to 48 weeks during the randomized placebo-controlled stage B)
研究者
研究点 (369)
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