ALS/MND Natural History Study Data Repository

NCT ID: NCT05966038

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

5000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-04-01

Study Completion Date

2030-12-31

Brief Summary

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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

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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.

Conditions

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ALS PLS MND (Motor Neurone DIsease) Kennedy Disease PMA - Progressive Muscular Atrophy PBP - Progressive Bulbar Palsy

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Henry Ford Health System

OTHER

Sponsor Role collaborator

Northwestern University

OTHER

Sponsor Role collaborator

Temple University

OTHER

Sponsor Role collaborator

St. Louis University

OTHER

Sponsor Role collaborator

University of Florida

OTHER

Sponsor Role collaborator

University of Minnesota

OTHER

Sponsor Role collaborator

Virginia Commonwealth University

OTHER

Sponsor Role collaborator

Fondazione Serena Onlus - Centro Clinico NeMO Milano

OTHER

Sponsor Role collaborator

Providence Health & Services

OTHER

Sponsor Role collaborator

University of Pittsburgh

OTHER

Sponsor Role collaborator

Istituti Clinici Scientifici Maugeri SpA

OTHER

Sponsor Role collaborator

Lahey Clinic

OTHER

Sponsor Role collaborator

Hadassah Medical Organization

OTHER

Sponsor Role collaborator

Tel Aviv Medical Center

OTHER

Sponsor Role collaborator

Kaiser Permanente

OTHER

Sponsor Role collaborator

Massachusetts General Hospital

OTHER

Sponsor Role lead

Responsible Party

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Alexander Sherman

Director, Center for Innovation and Bioinformatics

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Alexander Sherman

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

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Loma Linda University Health

Loma Linda, California, United States

Site Status RECRUITING

Kaiser Permanente

Los Angeles, California, United States

Site Status RECRUITING

University of Florida

Gainesville, Florida, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

Lahey Clinic

Burlington, Massachusetts, United States

Site Status RECRUITING

Henry Ford Health System

Detroit, Michigan, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Saint Louis University

St Louis, Missouri, United States

Site Status RECRUITING

Providence ALS Clinic

Portland, Oregon, United States

Site Status RECRUITING

Temple University Lewis Katz School of Medicine

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

Hadassah Medical Organization

Jerusalem, , Israel

Site Status RECRUITING

Tel Aviv Medical Center

Tel Aviv, , Israel

Site Status RECRUITING

Centro Clinico NEMO Milano

Milan, , Italy

Site Status RECRUITING

Istituti Clinici Scientifici Maugeri SpA

Milan, , Italy

Site Status RECRUITING

Countries

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United States Israel Italy

Central Contacts

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Natalia Tarasenko

Role: CONTACT

Phone: 1617240346

Email: [email protected]

Facility Contacts

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Chris Greer

Role: primary

Jennifer Steshyn

Role: primary

Emma Schmidt

Role: primary

Sorieba Fofanah

Role: primary

Melo Catia

Role: backup

Anne Vallis

Role: primary

Valerie Ferment

Role: primary

Katie Forsman

Role: primary

Tyler Regan

Role: primary

John Furey

Role: primary

Helen Ismail

Role: primary

Demetrius Carter

Role: primary

Michal Zabari

Role: primary

Beatrice Abramovich

Role: primary

Monica Montuori

Role: primary

Camilla Garrè

Role: primary

Study Documents

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Document Type: Individual Participant Data Set

View Document

Related Links

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Other Identifiers

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2013P001670

Identifier Type: -

Identifier Source: org_study_id

5U01FD008125-02

Identifier Type: FDA

Identifier Source: secondary_id

View Link

2022A011556

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2022A018800

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id