EUCTR2016-000942-77-DE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 Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease
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 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 (beta-human chorionic gonadotropin) at
- •-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 stopping and without an assistive device.
- •-The patient requires invasive-ventilation (non-invasive ventilation is
- •-The patient is not able to successfully perform repeated forced vital
- •capacity (FVC) measurements in upright position of =30% 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
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 122
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 17
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
相似试验
进行中（未招募）
1 期
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseasePompe disease (acid alpha-glucosidase deficiency)MedDRA version: 19.1 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850EUCTR2016-000942-77-DKGenzyme Corporation90
进行中（未招募）
1 期
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseasePompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850EUCTR2016-000942-77-ITGENZYME CORPORATIO90
进行中（未招募）
1 期
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseasePompe disease (acid alpha-glucosidase deficiency)MedDRA version: 24.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850EUCTR2016-000942-77-PTGenzyme Corporation100
进行中（未招募）
1 期
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseasePompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850EUCTR2016-000942-77-SEGenzyme Corporation100
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