An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naïve CRIM(-) Patients with Infantile-Onset Pompe Disease

Genzyme Corporation Inc0 个研究点目标入组 4 人开始时间: 2015年4月15日最近更新: 2015年7月10日

适应症

试验速览

暂无简介。

•The patient's legal guardian(s) must have provided written informed consent prior to any study-related procedures being performed
•The patient must have had a clinical diagnosis of Pompe disease as defined by documented acid alpha-glucosidase (GAA) deficiency (deficient endogenous GAA activity) in skin fibroblasts, muscle, or blood, or 2 GAA mutations. Consent was also sought from the biological parent(s) for parental GAA mutational analysis, but was not a requirement for study eligibility
•The patient must have not received Myozyme® or any rhGAA therapies prior to enrollment in the study
•The patient must be CRIM negative via Western Blot analysis performed on skin fibroblasts or via 2 known CRIM negative mutations (in which case CRIM status was to be confirmed by Western Blot analysis after enrollment)
•The patient's legal guardian(s) must have the ability to comply with the clinical protocol
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•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

•The patient had any medical condition that, in the opinion of the Investigator, could be exacerbated/precipitated by or interfere with the study regimen or assessments; such conditions may include but were not limited to human immunodeficiency virus, cancer, Hepatitis B, Hepatitis C, Cytomegalovirus, Herpes Simplex, John Cunningham (JC) virus, Parvovirus, or Epstein Barr virus or tuberculosis
•The patient had used any investigational product within 30 days prior to study enrollment
•The patient had or was required to have any live vaccination within 1 month prior to enrollment

发起方

Genzyme Corporation Inc