NL-OMON39502已完成4 期
A PHASE 4 PROSPECTIVE EXPLORATORY MUSCLE BIOPSY, BIOMARKER, AND IMAGING ASSESSMENT STUDY IN PATIENTS WITH LATE-ONSET POMPE DISEASE TREATED WITH ALGLUCOSIDASE ALFA. - Exploratory Muscle Biopsy Assessment Study (EMBASSY)
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Genzyme
- 入组人数
- 3
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
- 18 至 99（—）
入选标准
- •The patient is willing and able to provide signed informed consent.
- •The patient is >=18 years of age with confirmed acid a-glucosidase [GAA] enzyme deficiency from any tissue source and/or confirmed GAA gene mutations and without known cardiac hypertrophy.
- •The patient is able to ambulate a certain distance without stopping and without an assistive device. Use of assistive device for community ambulation is appropriate.
- •The patient has a certain forced vital capacity (FVC) in upright position.
- •The patient, if female and of childbearing potential, must have a negative pregnancy test at baseline. Note: All female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.
排除标准
- •The patient has had previous treatment with Enzyme Replacement Therapy.
- •The patient is wheelchair dependent.
- •The patient requires invasive-ventilation (non-invasive ventilation is allowed).
- •The patient is participating in another clinical study using investigational treatment.
- •The patient cannot submit to MRI examination because of a formal contraindication such as a pacemaker, implanted ferromagnetic metals, etc.
- •The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
研究者
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