Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease

EDISON PHARMACEUTICALS INC.0 个研究点开始时间: 2012年8月7日最近更新: 2013年1月7日

适应症

试验速览

暂无简介。

•1. Subjects with Diagnosis of Leigh syndrome who has previously received EPI-743 and continues to receive benefit from the use of EPI-743 2. Patient or parent’s or legal representative(s) able to consent and comply with protocol requirements 3. Subject (where appropriate, depending on age) and their parent(s) or legal representative(s) who have voluntarily signed the Informed Consent/Assent Form (ICF). Children will be informed about the program and asked to give assent (where appropriate, depending on age).
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Any condition, which in the opinion of the investigator could compromise the subject’s safety or adherence to treatment with EPI-743. 2. Previously demonstrated clinically significant allergy or hypersensitivity to with EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).

发起方