ALS/MND Natural History Study Data Repository
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease. Basic parameters: 18 years — 90 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Israel, Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Detailed description
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
* Longitudinal clinical and phenotypical data from routine clinical visits * Longitudinal clinical and historical phenotypical data transcribed from electronic health records and notes * Any other observational data that are of interest to the Investigator may be captured or linked to information in NeuroBANK.
Other Key Features
The site should track numbers of patients who declined participation. The site should have a mechanism in place to include an offer of participation to each patient in clinic (consenting does not require immediate data entry) Regular Data Quality checks may be performed at the site. Site personnel who intend to have access to NeuroBANK are trained and certified prior to given access.
Several registries, both cross-sectional and longitudinal, currently exist in ALS. NeuroBANK is distinct from other current registries in that it is a patient-centered platform that is designed to function as a data repository of patient data from clinical visits and multiple clinical research projects as well as linking these data to biorepository for tissue, imaging, and other biological information. This natural history study can act as a clinical research framework that may link clinical and clinical research data from current and past studies with biological specimens and image collections.
With obtained consents, biological specimens may be collected with bar-coded labels containing patient assigned study specific NeuroSTAmPs, printed from within NeuroBANK, and scanned into NeuroBANK's virtual distributed BioRepository module. Imaging studies are de-identified with NeuroSTAmPs and linked to clinical and phenotypical information. Anonymized clinical data obtained through this protocol are available to other researchers.
Study Population
Individuals who are seen during their clinical care visits are asked to allow their data to be uploaded and captured for clinical research. The NeuroBANK platform is located at and managed by the Center for Innovation and Bioinformatics (CIB), Neurological Clinical Research Institute (NCRI) of Massachusetts General Hospital (Boston, MA).
Consent Process
Every participant in the ALS clinic is approached to participate in this study. A signed informed consent form is obtained before any data are recorded for study purposes.
Primary outcome measures
- ALS Functional Rating Scale-Revised (ALSFRS-R) [Time frame: Every 3-4 months up to 5 years]
- Slow Vital Capacity (SVC) [Time frame: Every 3-4 months up to 5 years]
- Survival [Time frame: Every 3-4 months up to 5 years]
Secondary outcome measures (9)
- Vital Signs [Time frame: Every 3-4 months up to 5 years]
- Neurological Examination [Time frame: Through study completion, annually up to 5 years]
- El Escorial Criteria [Time frame: Through study completion, annually up to 5 years]
- Handheld Dynamometry [Time frame: Every 3-4 months up to 5 years]
- Grip Strength Testing [Time frame: Every 3-4 months up to 5 years]
- Rasch Overall ALS Disability Scale (ROADS) [Time frame: Every 3-4 months up to 5 years]
- 5 Times Sit to Stand [Time frame: Every 3-4 months up to 5 years]
- 10 Meter Walk Test [Time frame: Every 3-4 months up to 5 years]
- Physical Examination [Time frame: Through study completion, annually up to 5 years]
Eligibility criteria
Inclusion criteria
- 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)
Exclusion criteria
- Disease does not meet criteria for any motor neuron disorder
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
United States · 14 centers
- Loma Linda University Health — Loma Linda
- Kaiser Permanente — Los Angeles
- University of Florida — Gainesville
- Northwestern University — Chicago
- Lahey Clinic — Burlington
- Henry Ford Health System — Detroit
- Trinity Health Michigan — Grand Rapids
- University of Minnesota — Minneapolis
- … and 6 more centers
Israel · 2 centers
- Hadassah Medical Organization — Jerusalem
- Tel Aviv Medical Center — Tel Aviv
Italy · 2 centers
- Centro Clinico NEMO Milano — Milan
- Istituti Clinici Scientifici Maugeri SpA — Milan
Identifiers
NCT: NCT05966038 · 2013P001670 · 5U01FD008125-02 · 2022A011556 · 2022A018800