A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III

Zymenex A/S0 个研究点目标入组 20 人开始时间: 2012年8月6日最近更新: 2015年7月10日

适应症

试验速览

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•1.Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
•2.The subject and his/her guardian(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 < 10% of normal activity (historical data)
•4.The subject must have an age at the time of screening = 5 years and = 35 years
•5.The subject must have the ability to physically and mentally cooperate in the tests
•6.The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 10
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1.The subjects diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity
•2.The subject cannot walk without support
•3.Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
•4.History of BMT
•5.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
•6.Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
•7.Pregnancy: Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
•8.Psychosis within the last 3 months
•9.Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
•10.Participation in other interventional trials testing IMP within the last 3 months

发起方