ASSESS ALL ALS - Longitudinal Biomarker Study for Symptomatic ALS and Control Participants
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2,000
- 试验地点
- 55
- 主要终点
- ALS Functional Rating Scale-Revised (ALSFRS-R)
研究概览
简要总结
The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is being funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases.
ASSESS protocol is specific for symptomatic ALS and control participants. This protocol includes both on-site and off-site(remote) participants. The participants will be followed for 24 months (2 years), and will include collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once a month. Participants who are coming into clinic may also provide optional Cerebrospinal Fluid (CSF) samples.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •for ALS participants:
- •Age 18 years or older
- •Capable of providing informed consent
- •Willing to follow study procedures
- •Diagnosis of ALS by a physician
- •Access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient)
- •Inclusion Criteria for control participants:
- •Age 18 years or older
- •Capable of providing informed consent
- •Willing to follow study procedures
- •No diagnosis of ALS , Progressive Muscular Atrophy (PMA) or Primary Lateral Sclerosis (PLS)
- •No history of familial ALS/Frontotemporal Dementia (FTD) in a close family member** unless the participant has previously tested negative for the known causative ALS genes. Participants with a family history of singleton ALS are permitted to enroll.
- •** Defined by the presence of a known ALS causative gene such as C9orf72 in a family member or a family history suggestive of an inherited ALS/FTD syndrome defined by two family members with a history of ALS and/or FTD.
- •Access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient)
排除标准
- •for all participants:
- •Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, active suicidal ideation, suicide attempt, or untreated major depression <= 90 days of screening, that would interfere with the study procedure, according to Investigator's judgement.
- •Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, malignant and potentially progressive cancer) that would render the participant unlikely to be able to complete 12 months of follow-up, according to Investigator's judgment.
- •Exclusion Criteria for participants undergoing optional Lumbar Puncture
- •Medically unable to undergo lumbar puncture (LP) as determined by the site investigator (i.e., bleeding disorder, a skin infection at or near the LP site, known or suspected intracranial or intraspinal tumor or other cause of increased intracranial pressure).
- •Allergy to Lidocaine or other local anesthetic agents.
- •Use of anticoagulant medication or antiplatelet medications (aside from aspirin 81 mg) that cannot be safely withheld prior to lumbar puncture.
- •Blood dyscrasia, abnormal bleeding diathesis, or the use of dialysis for renal failure.
- •Current pregnancy based on participant self-report
- •Clinical judgement of the site investigator that the participant would be unable to undergo multiple lumbar punctures.
研究组 & 干预措施
Symptomatic ALS on-site participants
Symptomatic ALS Participants who are completing on-site visits at one of the 35 participating ALL ALS sites.
Symptomatic ALS off-site (remote) participants
Symptomatic ALS Participants who are completing visits study visits remotely through video-conferencing. Home Phlebotomy is being used to collect blood from these participants.
Control participants
Control participants who do not have a diagnosis of ALS , Progressive Muscular Atrophy (PMA) or Primary Lateral Sclerosis (PLS). These participants complete on-site visits.
结局指标
主要结局
ALS Functional Rating Scale-Revised (ALSFRS-R)
时间窗: Baseline, 4, 8, 12, 16, 20 and 24 months for ALS participants only.
Questionnaire administered by a clinician that includes a series of questions about participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
ALS Functional Rating Scale-Revised Self entry (ALSFRS-RSE)
时间窗: Baseline, 2, 4 ,6, 8, 10, 12, 14,16, 18,20, 22 and 24 months for ALS participants only
Questionnaires completed by participants that includes questions about participants ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
次要结局
- ALS Impairment Multidomain scale (AIMS)(1, 3, 5,7,9,11,13,15,17,19,21 and 23 months from baseline for ALS participants only)
- Social Determinants of Health(Months 3 and 4 for all cohorts)
- Environmental History(Month 1 for all cohorts)
- Cognitive assessment (ECAS)(Baseline, 12 and 24 months for all cohorts)
- Vital Capacity(Baseline, 4,8,12,16,20 and 24 months for on-site ALS participants; Baseline, 12 and 24 months for control participants)
- Digital Speech Assessment(Baseline, and monthly (at month 1,2, 3 through month 24 from baseline))
- HHD of 3 hand muscles bilaterally(Baseline, 4,8,12,16,20 and 24 months for on-site ALS participants; Baseline, 12 and 24 months for control participants)