ALS/MND Natural History Study Data Repository
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 5,000
- 试验地点
- 34
- 主要终点
- ALS Functional Rating Scale-Revised (ALSFRS-R)
研究概览
简要总结
This is a data repository for multi-site multi-protocol clinic-based Natural History Study of ALS and Other Motor Neuron Disorders (MND).
All people living with ALS or other MNDs who attend clinics at the Study hospitals (sites) are offered to participate in the Study.
The Sites collect so-called Baseline information including demographics, disease history and diagnosis, family history, etc. At each visit, the Sites also collect multiple disease-specific outcome measures and events. The information is captured in NeuroBANK, a patient-centric clinical research platform.
The Sites have an option to choose to collect data into 20+ additional forms capturing biomarkers and outcome measures. Captured data after its curation are anonymized (all personal identifiers and dates are being removed), and the anonymized dataset is shared with medical researchers via a non-exclusive revocable license.
Funding Source - Biogen, Inc.; Mitsubishi Tanabe Pharma America; FDA OOPD.
详细描述
Most people with ALS or MND who are seen clinically by the PI or PI designees at participating sites will be recruited into this study. This well-established framework provides an opportunity to offer to a large proportion of people with ALS/MND a chance to participate in a clinical research project.
Upon obtaining consent, the following is done:
People with ALS/MND are assigned a Neurological Global Unique Identifier (NeuroGUID) and its study-specific derivative, NeuroSTAmP, which is used to link this Study's information to biological samples, images, and clinical data obtained from those individuals in this Study and other studies/trials, in which these patients participate(d).
The following information is captured in NeuroBANK platform:
Baseline information
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A clinical diagnosis of El Escorial of suspected, possible, probable, or definite ALS or
- •Other motor neuron disorders, including but not limited to Spinal-Bulbar Muscular Atrophy (SBMA, Kennedy's disease), Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis (PLS), Progressive Muscular Atrophy (PMA), and Progressive Bulbar Palsy (PBP)
排除标准
- •Disease does not meet criteria for any motor neuron disorder
结局指标
主要结局
ALS Functional Rating Scale-Revised (ALSFRS-R)
时间窗: Every 3-4 months up to 5 years
The ALS Functional Rating Scale revised is a 12-item rating scale that measures the progression of disability in patients with ALS. The ALSFRS-r assesses patients' levels of self-sufficiency in areas of feeding, grooming, ambulation and communication. The higher the score, the earlier in the disease progression. It consists of 12 questions with a maximum score of 48 points.
Slow Vital Capacity (SVC)
时间窗: Every 3-4 months up to 5 years
Slow vital capacity test normalized to age, sex, and height
Survival
时间窗: Every 3-4 months up to 5 years
Patient survival or survival equivalent (time to ventilator or feeding tube). Survival equivalent is assessed during routine clinical visits, while mortality event may be reported when becomes known to the treating clinician..
次要结局
- Rasch Overall ALS Disability Scale (ROADS)(Every 3-4 months up to 5 years)
- Vital Signs(Every 3-4 months up to 5 years)
- Handheld Dynamometry(Every 3-4 months up to 5 years)
- 10 Meter Walk Test(Every 3-4 months up to 5 years)
- Grip Strength Testing(Every 3-4 months up to 5 years)
- 5 Times Sit to Stand(Every 3-4 months up to 5 years)
- Physical Examination(Through study completion, annually up to 5 years)
- Neurological Examination(Through study completion, annually up to 5 years)
- El Escorial Criteria(Through study completion, annually up to 5 years)
研究者
Alexander Sherman
Director, Center for Innovation and Bioinformatics
Massachusetts General Hospital