EUCTR2016-000942-77-SE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
- 100
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
- 性别
- All
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 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