NCT01285700Unknown2 期
A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety 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
- 2 期
- 发起方
- Zymenex A/S
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Reduction of Oligosaccharides in urine
研究概览
简要总结
This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.
研究设计
- 研究类型
- Interventional
- 分配方式
- 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 21 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 ≤ 21 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 25
Active Comparator
25 U/kg
干预措施: Lamazym (Drug)
Lamazym 50
Active Comparator
50 U/kg
干预措施: Lamazym (Drug)
结局指标
主要结局
Reduction of Oligosaccharides in urine
时间窗: 3 months (interim evaluation) + 6 months
Efficacy endpoint evaluation as change from baseline
次要结局
- Reduction of Oligosaccharides in serum(3 months (interim evaluation) + 6 months)
- Reduction of Oligosaccharides in CSF(3 months (interim evaluation) + 6 months)
- The distance walked in 6 minutes(3 months (interim evaluation) + 6 months)
- The number of steps climbed in 3 minutes(3 months (interim evaluation) + 6 months)
- Pulmonary Function(3 months (interim evaluation) + 6 months)
- Adverse events(1 week)
- Development of clinically significant changes in vital signs and change in physical examination(1 week)
- Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)(4 weeks)
- Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies(2 weeks)
研究者
研究点 (1)
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