NCT01681940已完成2 期
A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of 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
- 状态
- 已完成
- 发起方
- Zymenex A/S
- 入组人数
- 10
- 试验地点
- 4
- 主要终点
- Reduction of Oligosaccharides in blood serum
研究概览
简要总结
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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 subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
- •Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- •The subject and his/her guardian(s) must have the ability to comply with the protocol
排除标准
- •The subject 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
- •Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial.
- •Pregnancy: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study
- •Psychosis within the last 3 months
- •Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
- •Participation in other interventional trials testing IMP except for studies with Lamazym
研究组 & 干预措施
Lamazym
Experimental
1 mg/kg body weight
干预措施: Lamazym (Drug)
结局指标
主要结局
Reduction of Oligosaccharides in blood serum
时间窗: 6 months
Efficacy endpoint evaluation as change from baseline
次要结局
- Reduction of Oligosaccharides in CSF(6 months)
- Adverse events(1 week)
- Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)(1 week)
- Development of clinically significant changes in vital signs and change in physical examination(1 week)
- The number of steps climbed in 3 minutes (3-minute stair climb)(6 months)
- The distance walked in 6 minutes (6-minute walk test)(6 months)
- Pulmonary function(6 months)
- Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies(1 week)
研究者
研究点 (4)
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