NCT01268358已完成1 期
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
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Zymenex A/S
- 入组人数
- 10
- 主要终点
- To evaluate the safety profile of rhLAMAN (Lamazym)
研究概览
简要总结
This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.
It is the hypothesis that Lamazym is safe to use.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
- •The patient must have an age at the time of screening ≥ 5 year and ≤ 20 years
- •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).
- •The patient must have the ability to mentally cooperate in the cognitive and motor function tests
- •The patient must have the ability to hear and follow a request. Hearing aids can be worn.
- •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)
- •The patient and his/her guardian(s) must have the ability to comply with the protocol
排除标准
- •The patient cannot walk without support.
- •Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
- •History of bone marrow transplantation
- •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
- •Presence of an ECHO with abnormalities within half a year that, in the opinion of the Investigator, would preclude participation in the trial
- •Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial
- •Psychosis within the last 3 months
研究组 & 干预措施
Lamazym 6.25
Experimental
干预措施: Lamazym (Drug)
Lamazym 12.5
Experimental
干预措施: Lamazym (Drug)
Lamazym 25
Experimental
干预措施: Lamazym (Drug)
Lamazym 50
Experimental
干预措施: Lamazym (Drug)
Lamazym 100
Experimental
干预措施: Lamazym (Drug)
结局指标
主要结局
To evaluate the safety profile of rhLAMAN (Lamazym)
时间窗: 1-5 weeks
次要结局
- To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03)(1 week)
- To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma(1 dosis)
研究者
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