Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS, Amyotrophic Lateral Sclerosis, biomarker, observational
Brief summary
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.
Interventions
None listed
Sponsors
St. Joseph's Hospital and Medical Center, Phoenix
Massachusetts General Hospital
National Institute of Neurological Disorders and Stroke (NINDS)
Foundation for the National Institutes of Health
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
for ALS participants: 1. Age 18 years or older 2. Capable of providing informed consent 3. Willing to follow study procedures 4. Diagnosis of ALS by a physician 5. 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: 1. Age 18 years or older 2. Capable of providing informed consent 3. Willing to follow study procedures 4. No diagnosis of ALS , Progressive Muscular Atrophy (PMA) or Primary Lateral Sclerosis (PLS) 5. 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. 6. 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)
Exclusion criteria
for all participants: 1. 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. 2. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ALS Functional Rating Scale-Revised (ALSFRS-R) | 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) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Questionnaires completed by participants that assess severity of the ALS disease |
| Social Determinants of Health | Months 3 and 4 for all cohorts | Questionnaires completed by participants that includes questions about non-medical factors that affect health outcomes |
| Environmental History | Month 1 for all cohorts | Questionnaires completed by participants that includes questions about environmental exposures such as chemicals, smoking, alcohol etc. |
| Cognitive assessment (ECAS) | Baseline, 12 and 24 months for all cohorts | ECAS is a multi-domain assessment administered by study staff, to evaluate different aspects of cognitive function |
| Vital Capacity | Baseline, 4,8,12,16,20 and 24 months for on-site ALS participants; Baseline, 12 and 24 months for control participants | Change in respiratory function over time as measured by Slow Vital Capacity (SVC). |
| Digital Speech Assessment | Baseline, and monthly (at month 1,2, 3 through month 24 from baseline) | Participants will be asked to record voice samples at home once a month using a digital application installed on their device. |
| 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 | Hand-held dynamometry (HHD) will be used as a quantitative measure of muscle strength for the three intrinsic hand muscles, bilaterally |
Countries
Puerto Rico, United States
Contacts
CONTACTALL ALS Patient Navigator
Outcome results
None listed