EUCTR2010-022084-36-DK进行中（未招募）不适用
A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis.
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Zymenex A/S
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.The patient must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
- •2.The patient must have an age at the time of screening = 5 year and = 20 years
- •3.The patient must have physical ability to perform 6-minutes walk test (6MWT), 3 minute-stair climb test (3MSCT) and pulmonary lung function test (spirometry, body plethysmography).
- •4.The patient must have the ability to mentally cooperate in the cognitive and motor function tests
- •5.The patient must have the ability to hear and follow a request. Hearing aids can be worn.
- •6.Patient or patient’s legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- •7.The patient and his/her guardian(s) must have the ability to comply with the protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •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
排除标准
- •1.The patient cannot walk without support.
- •2.Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
- •3.History of bone marrow transplantation
- •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.Presence of an ECHO with abnormalities within half a year 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
研究者
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