Clinical Extension Study for Assessing the Safety and Efficacy of the Intravenous Administration of Cellavita-HD in Huntington's Disease Patients Who Participated in the ADORE-DH Study.
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 35
- 试验地点
- 2
- 主要终点
- Maintenance of effectiveness on clinical progression of the disease
研究概览
简要总结
Cellavita-HD is a stem-cell therapy for Huntington's Disease. Open label, single treatment, extension study for long-term safety and efficacy evaluation of Cellavita-HD intravenous administration in Huntington's disease patients who participated of ADORE-DH trial.
详细描述
This extension study will include the subjects who participated the study phase II for dose-response evaluation of Cellavita-HD intravenous administration in Huntington's disease patients (maximum 35 subjects). Because it is a extension study, with an open scenario, the subjects will receive the maximum dose tested in the last clinical trial (ADORE-DH), corresponding to 6 x 10^6 cells/weight range for each treatment cycle planned. Throughout of 24 months, the subjects will receive total dose planned divided into three administrations per cycle (2 x 10^6 cells/weight range), that each will occur every 30 days. The maintaining the effectiveness of intravenous administration of Cellavita-HD product over time on clinical progression Huntington's disease will be verified by comparing the UHDRS total score at the end of the 24th study month against baseline score. Secondary evidences of efficacy will be evaluated by through the data of functional state, total functional capacity, functional independence, psychiatric symptoms and cognition from UHDRS scale. Additionally, related data to clinical worsening, change of Body Mass Index (BMI), risk of suicide attempt and neurological image improvement will be evaluated. Safety evaluation will included the incidence and classification of the adverse events experienced by the subjects during the study.
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
- 21 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Phase II study participants (ADORE-DH) who express interest in participating in the extension period and who may have significant clinical benefits (motor, cognitive, behavioral and functional capacity) at the Investigator's judged after intravenous application of the Cellavita-HD product;
- •Provide consent by signing in two copies of the Informed Consent Form;
- •Participant using an acceptable contraceptive method.
排除标准
- •History of malignant neoplasia;
- •Present any clinical and laboratory condition or comorbidity that, at the physician's judged, may endanger the health of the research participant and prevent him / her from being part of the extension study;
- •known hypersensitivity to the investigational product and / or products of bovine origin;
- •Research participant who has participated in clinical trial protocols in the last twelve (12) months, unless the Investigator believes that there may be direct benefit to it (Resolution CNS 251 of August 7, 1997, item III, sub-item J);
- •Pregnant or lactating participant;
- •In use or anticipated use of immunosuppressive drugs as well as any other drug in which the use is restricted to this research protocol.
结局指标
主要结局
Maintenance of effectiveness on clinical progression of the disease
时间窗: Two years (every study visit)
The maintenance of treatment effectiveness will be verified by comparing the total UHDRS (Unified Huntington's Disease Rating Scale) score registered at the end of the 24th month of study in relation to the baseline score.
次要结局
- BMI assessment(Two years)
- Risk of suicidal ideation and/or behavior by C-SSRS(Two years)
- CNS assessment by MRI (Magnetic Ressonance Imaging)(One year)
- Risk of suicidal ideation and/or behavior by HDS(Two years)
- Clinical neurological worsening over the treatment by UHDRS(Two years)