2024-514773-22-00招募中4 期
A French multicenter Phase 4 open label extension study of long-term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 10
- 主要终点
- Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death
研究概览
简要总结
To assess long-term safety in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France, or until September 2026, whichever comes first.
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Patient with LOPD or IOPD who has previously completed Study EFC14028, LTS13769, or ACT14132 in France, and reimbursement for avalglucosidase alfa is not yet granted in France.
- •The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, 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 result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
- •Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception.
排除标准
- •Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients.
- •Patient who permanently discontinued avalglucosidase alfa in a previous clinical study
- •Pregnant or breastfeeding female patient
- •The patient is concurrently participating in another clinical study of investigational treatment
- •The patient, in opinion of the Investigator, is unable to comply with the requirements of the study
- •The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatobiliary, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.
- •Individuals accommodated in an institution because of regulatory or legal order; prisoners, or patients who are legally institutionalized.
结局指标
主要结局
Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death
Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death
次要结局
- Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants
- Assessment of quick motor function test (QMFT) for LOPD participants
- Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants
- Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants
- Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants
- Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants
- Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants
- PedsQL score for IOPD participants
- Left Ventricular Mass Index (LVMI) Z-score in IOPD participants
研究者
Clinical Sciences and Operations
Scientific
Sanofi Winthrop Industrie
研究点 (10)
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