NCT04929236招募中3 期
Multicenter, Prospective, Double-Blinded, Parallel Group, Randomized Phase III Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Octapharma
- 入组人数
- 30
- 试验地点
- 7
- 主要终点
- Change in CIDP Baseline
研究概览
简要总结
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
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)
盲法说明
Double-Blinded
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥2 years and ≤17 years.
- •Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone [1]
- •Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤
- •Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by the patient, if age appropriate per Independent Ethics Committee [IEC]/Institutional Research Board [IRB] requirements).
排除标准
- •Patients with previously diagnosed CIDP who lack any CIDP symptoms.
- •Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
- •Patients who have previously failed immunoglobulin therapy for CIDP.
- •Patients who received immunoglobulin or plasma exchange (PEX) within eight weeks prior to the Baseline Visit (washout phase). However, if a patient has clinical evidence of confirmed CIDP relapse during the washout phase (consistent with an increase in mRS of ≥1), they are eligible for trial enrolment.
- •Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
- •Patients on unstable (change in prescribed dose within the last eight weeks) corticosteroids or rituximab use.
- •Patients with known or suspected hypersensitivity, anaphylaxis, or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
- •Female patients who are breastfeeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method while on the study (acceptable methods of birth control for this study include: intrauterine device [IUD], hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap).
- •Presence of medical history information or clinical symptoms suggestive of human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.
- •Severe liver and/or kidney disease (alanine aminotransferase [ALT] > 3 × upper limit of normal [ULN]; aspartate aminotransferase [AST] > 3 × ULN; and/or creatinine levels >44 µmol/L for children ages 2-3 years, >62 µmol/L for children ages 4-10 years, and >89 µmol/L for children ages 11-17 years.
- •Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
- •History of alcohol or drug abuse in the previous year, per Investigator's opinion.
- •Unable or unwilling to comply with the study protocol.
- •Receipt of any other investigational medicinal product (IMP) within three months before study entry or participating in another interventional clinical study. Prior participation in an observational or open-label study involving an approved product may be allowed but require prior consultation with the Medical Monitor to assess eligibilty.
- •Any other condition(s) that, in the Investigator's opinion, makes it undesirable for the patient to participate in the study or may interfere with protocol compliance.
研究组 & 干预措施
Panzyga Low Dose
Experimental
1.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages.
干预措施: Panzyga (Drug)
Panzyga High Dose
Experimental
2.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages.
干预措施: Panzyga (Drug)
结局指标
主要结局
Change in CIDP Baseline
时间窗: Up to 24 weeks
Evaluate the efficacy of two PANZYGA dose regimens in pediatric CIDP patients based on change in CIDP symptoms, measured by the Modified Rankin Score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
次要结局
- Time to CIDP Relapse(Up to 24 weeks)
- CIDP Relapse(Up to 24 weeks)
- Percentage of Patients With Good/Excellent Response(Up to 24 weeks)
研究者
研究点 (7)
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