NCT01681953已完成3 期
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.
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Zymenex A/S
- 入组人数
- 25
- 试验地点
- 6
- 主要终点
- The number of steps climbed in 3 minutes (3-minute stair climb test)
研究概览
简要总结
The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age 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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
- •The subject and his/her guardian(s) must have the ability to comply with the protocol
- •The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-Mannosidase activity < 10% of normal activity (historical data)
- •The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
- •The subject must have the ability to physically and mentally cooperate in the tests
- •The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
排除标准
- •The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity < 10% of normal activity
- •The subject cannot walk without support
- •Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
- •History of BMT
- •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: Pregnant woman is excluded. 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
- •Psychosis; any psychotic disease, also in remission, is an exclusion criteria
- •Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
- •Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
- •Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
- •Total IgE >800 IU/ml
- •Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
研究组 & 干预措施
Lamazym
Active Comparator
1 mg Lamazym/kg body weight
干预措施: Lamazym (Drug)
Placebo
Placebo Comparator
Placebo is formulated as an isotonic phosphate buffer with glycine and mannitol
干预措施: Placebo (Drug)
结局指标
主要结局
The number of steps climbed in 3 minutes (3-minute stair climb test)
时间窗: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks
Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
Reduction of oligosaccharides in serum
时间窗: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks
Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
次要结局
- Development of clinically significant changes in vital signs and change in physical examination(1 week)
- Clinical laboratory parameters (hematology, biochemistry and urinalysis)(1 week)
- Forced Vital Capacity(Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks)
- The distance walked in 6 minutes (6-minute walk test)(Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks)
- Development of Lamazym antibodies and neutralizing/inhibitory antibodies(1 week)
- Adverse Events(1 week)
研究者
研究点 (6)
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