NL-OMON46174已完成3 期
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease - COMET
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 2
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •-The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations
- •-The patient and/or their parent/legal guardian is willing and able to provide signed informedconsent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
- •-The patient (and patient*s legal guardian if patient is <18 years of age) must have the ability to
- •comply with the clinical protocol.
- •-The patient, if female and of childbearing potential, must have a negative pregnancy test (betahuman
- •chorionic gonadotropin) at baseline.
排除标准
- •-The patient is younger than 3 years of age.;-The patient has known Pompe specific cardiac hypertrophy.;-The patient is wheelchair dependent.;-The patient is not able to ambulate 40 meters (approximately 130 feet) without topping and without an assistive device.;-The patient requires invasive-ventilation (non-invasive ventilation is allowed).;-The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of >=40% predicted and <=85% predicted.;-The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.;-The patient has prior or current use of immune tolerance induction therapy.
研究者
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