Open-Label, Exploratory Study of the Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)
试验速览
- 阶段
- 2 期
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Change in Soluble IL-2 Receptor level in peripheral blood
研究概览
简要总结
An open-label, non-randomised, uncontrolled, proof-of-concept study of patients with HTLV-I-associated myelopathy/Tropical Spastic Paraparesis (HAM/TSP). Participants will receive oral administration of tamibarotene in the amount of 2 mg daily over a period of 12 weeks, then 4mg daily for another 12 weeks. The patients will be followed up for further 8 weeks. Efficacy will be monitored by measuring clinical scores including motor and urination function, HTLV-1 proviral load, immunological parameters, and markers in the spinal fluid. Safety will be evaluated at the same time.
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
- 30 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have been diagnosed as HAM according to the WHO criteria
- •Patients who are positive for HTLV-I antibody in the spinal fluid
- •Patients, if female, who are not pregnant or breastfeeding, either agreed to take contraceptive measures during and two years after the treatment, or sterile
- •Patients, if male, who agreed to take contraceptive measures during and six months after the treatment
- •Patients who have been informed and understood the contents of the study and consented to participate in the signed form.
排除标准
- •Patients who has a rapid progress in the symptoms defined as an increase of two or more in Osame's Motor Disability Score for HAM patients in the past one year.
- •Patients of hyperlipidemia (serum triglyceride higher than 400 mg/dL)
- •Patients who were administered new or increased dose of corticosteroid in the past 8 weeks before the intervention
- •Patients who received steroid pulse therapy in the past 8 weeks before the intervention
- •Patients who were administered new or increased dose of immunosuppressant in the past 8 weeks before the intervention
- •Patients with a history of serious drug allergy
- •Patients with significant complication such as malignancy, severe heart failure, and other serious diseases.
- •Patients who were in the past administered etretinate.
结局指标
主要结局
Change in Soluble IL-2 Receptor level in peripheral blood
时间窗: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
Change in HTLV-I viral load in peripheral blood
时间窗: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks
Change in T cell population in peripheral blood
时间窗: 0,12, 24, 28 and 32 weeks
Change in cerebrospinal fluid examination
时间窗: baseline and after the treatment defined as from 24 to 32 weeks
次要结局
- Change in Osame's Motor Disability Score for HAM patients(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)
- Change in The Expanded Disability Status Scale (EDSS)(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)
- Change in timed 10m walk(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)
- Change in Manual Muscle Testing and vibratory perception of the lower limbs(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)
- Change in Modified Ashworth Scale(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)
- Change in Urination function and defecation score(0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks)