Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.
试验速览
- 阶段
- 3 期
- 入组人数
- 1
- 主要终点
- Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.
研究概览
简要总结
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease.
详细描述
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis).
The patient was randomized to be treated with intravenous immunoglobulin or placebo 1:1 (crossover) once a month for at least one year.
Main objective: improvement of the action myoclonus. Secondary objectives: Improvement in the overall score and in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial.
The first analyst was scheduled at one year from the start of the trial. The program was to discuss the patient's analysis data and to let the patient decide in three possible ways: to continue the trial, to continue treatment with immunoglobulins, to suspend the treatment. Depending on the decision, it was planned to follow the patient throughout the year after the analysis, at least for one year.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Drug and placebo had the same appearance and are wrapped in foil paper before being shown to the patient.
Outcome evaluation is carried out by personnel not involved in the treatment at a distant site, one month after treatment.
入排标准
- 年龄范围
- 18 Years 至 25 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Malattia di Unverricht-Lundborg (genetic diagnosis)
排除标准
- •Contraindications to intravenous immunoglobulin
研究组 & 干预措施
Immunoglobulin
Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.
干预措施: Intravenous immunoglobulin (Drug)
Saline solution
Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.
干预措施: Intravenous immunoglobulin (Drug)
结局指标
主要结局
Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.
时间窗: monthly for one year
The range for Action Myoclonus Score is 0 (best) - 160 (worst, , i.e. more severe involuntary movements). Percent change = 100 X (Placebo UMRS4 - Treatment UMRS4) / Placebo UMRS4).
次要结局
- Unified Myoclonus Rating Scale (UMRS) overall score improvement.(monthly for one year)
- Patient's preference(one year)
研究者
Alfonso Ciccone
MD
Azienda Socio Sanitaria Territoriale di Mantova