A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis - A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy

CHIESI FARMACEUTICI S.P.A.0 个研究点目标入组 3 人开始时间: 2020年11月5日最近更新: 2020年11月16日

适应症

试验速览

暂无简介。

•1)Patient’s custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities;
•2)The subject’s custodial parent(s) must have the ability to comply with the protocol;
•3)The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity in leukocytes or fibroblasts < 10% of normal activity (historical data);
•4)The subject must have an age at the time of screening < 6 years.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•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) The subject’s diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity;
•2)Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis;
•3)History of Bone Marrow Transplantation (BMT);
•4)Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial;
•5)Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial;
•6)Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial;
•7)Participation in other interventional trials testing the IMP within the last 3 months.

发起方