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 - PTA Avalglucosidase

Sanofi-Aventis Recherche & Développement0 个研究点目标入组 18 人开始时间: 2021年8月16日最近更新: 2024年8月12日

适应症

试验速览

暂无简介。

•- Patient with Pompe Disease who has previously completed Study EFC14028, LTS13769, or ACT14132 of avalglucosidase alfa studies 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 (ß-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.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 13
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•Participants are excluded from the study if any of the following criteria apply:
•- Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients.
•- The patient is concurrently participating in another clinical study of investigational treatment.
•- 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.

发起方