ASSESS ALL ALS study
Cynthia Bodkin, MD
Primary Investigator
Brief description of study
The primary goal for this protocol is to establish a robust clinical trial-like infrastructure to coordinate recruitment, enrollment, and longitudinal follow up of a cohort of people with ALS and associate control participants. The overarching goal of the consortium is to prospectively collect, store and harmonize a large set of longitudinal data, which will be shared through the AMP ALS central portal, and biospecimens that will be linked to the clinical data for open science sharing with the research community.
Detailed description of study
This protocol is specific for symptomatic ALS and control participants. This study is designed to be inclusive of all people who have been diagnosed with ALS and control participants who are reasonably likely to be able to provide longitudinal data. Study visits will be conducted during routine clinical visits or as stand-alone research visits, with either a combination of in-person and remote visits or entirely remote visits, an approach that has been successful on a smaller scale in prior observational studies.
- Establish the necessary infrastructure, policies, and procedures to recruit a significant portion of people with ALS in the US into the ALL ALS study, utilizing both in-person and remote participation methods.
- Enroll participants into ALL ALS, utilizing a variety of in person and remote strategies to ensure that principles of diversity, equity, and inclusion are rigorously adhered to.
- Follow participants for 2 years, measuring a wide range of ALS outcome measures and gather long term survival data.
- Associated with clinical data collection, collect a robust set of biofluids for investigation of new markers and to further understand the relationship of existing biomarkers to clinical status. Biofluid samples will be obtained at
study site visits, and via home visits for remote participants. Biofluids will include longitudinal collection of whole blood, plasma, serum, and cerebrospinal fluid (on a subset of participants). Additional biosample types may be collected in collaboration with AMP ALS input.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Healthy, Amyotrophic Lateral Sclerosis, ALS
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Age: 18 years - 100 years
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Gender: All
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 member1
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.
1 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.
Exclusion Criteria for participants undergoing optional Lumbar Puncture
1. 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).
2. Allergy to Lidocaine or other local anesthetic agents.
3. Use of anticoagulant medication or antiplatelet medications (aside from aspirin 81 mg)
that cannot be safely withheld prior to lumbar puncture.
4. Blood dyscrasia, abnormal bleeding diathesis, or the use of dialysis for renal failure.
5. Current pregnancy based on participant self-report
6. Clinical judgement of the site investigator that the participant would be unable to undergo
multiple lumbar punctures.
