EUCTR2016-000942-77-DK进行中（未招募）1 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 Treatmentnaïve Patients with Late-onset Pompe Disease
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 90
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •-The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
- •-The patient must provide signed informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is(are) required for legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from patients, if applicable.
- •-The patient (and patient’s legal guardian if patient is legally minor as defined by local regulation) 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 2
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 94
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •-The patient is <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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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 Diseaseacid alfa glucosidase deficiency10021605Pompe DiseaseNL-OMON46174Sanofi-aventis2