ALS/MND Natural History Study Data Repository

NCT05966038 | Recruiting

• 4 other identifiers: 2013P0016705U01FD008125-022022A0115562022A018800
• Study Type: observational
• Target: 5,000
• Locations: 3 countries
• Sites: 16

Brief Summary

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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• ALS Functional Rating Scale-Revised (ALSFRS-R)

  The ALS Functional Rating Scale revised is a 12-item rating scale that measures the progression of disability in patients with ALS. The ALSFRS-r assesses patients' levels of self-sufficiency in areas of feeding, grooming, ambulation and communication. The higher the score, the earlier in the disease progression. It consists of 12 questions with a maximum score of 48 points.

  Every 3-4 months up to 5 years

• Slow Vital Capacity (SVC)

  Slow vital capacity test normalized to age, sex, and height

  Every 3-4 months up to 5 years

• Survival

  Patient survival or survival equivalent (time to ventilator or feeding tube). Survival equivalent is assessed during routine clinical visits, while mortality event may be reported when becomes known to the treating clinician..

  Every 3-4 months up to 5 years

Secondary Outcomes (9)

• Vital Signs

  Every 3-4 months up to 5 years

• Neurological Examination

  Through study completion, annually up to 5 years

• El Escorial Criteria

  Through study completion, annually up to 5 years

• Handheld Dynamometry

  Every 3-4 months up to 5 years

• Grip Strength Testing

  Every 3-4 months up to 5 years

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All people living with ALS/MND at participating multidisciplinary ALS/MND clinics

You may qualify if:

• 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)

You may not qualify if:

• Disease does not meet criteria for any motor neuron disorder

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Henry Ford Health System: collaborator
• Northwestern University: collaborator
• Temple University: collaborator
• St. Louis University: collaborator
• University of Florida: collaborator
• University of Minnesota: collaborator
• Virginia Commonwealth University: collaborator
• Fondazione Serena Onlus - Centro Clinico NeMO Milano: collaborator
• Providence Health & Services: collaborator
• University of Pittsburgh: collaborator
• Istituti Clinici Scientifici Maugeri SpA: collaborator
• Lahey Clinic: collaborator
• Hadassah Medical Organization: collaborator
• Tel Aviv Medical Center: collaborator
• Kaiser Permanente: collaborator

Study Sites (16)

Loma Linda University Health

Loma Linda, California, 92354, United States

RECRUITING

Kaiser Permanente

Los Angeles, California, 90027, United States

RECRUITING

University of Florida

Gainesville, Florida, 32610, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

Lahey Clinic

Burlington, Massachusetts, 01805, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Saint Louis University

St Louis, Missouri, 63104, United States

RECRUITING

Providence ALS Clinic

Portland, Oregon, 97213, United States

RECRUITING

Temple University Lewis Katz School of Medicine

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, 15360, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 22908, United States

RECRUITING

Hadassah Medical Organization

Jerusalem, 9112001, Israel

RECRUITING

Tel Aviv Medical Center

Tel Aviv, 64239, Israel

RECRUITING

Centro Clinico NEMO Milano

Milan, 20162, Italy

RECRUITING

Istituti Clinici Scientifici Maugeri SpA

Milan, 20138, Italy

RECRUITING

Related Links

• ALS/MND NATURAL HISTORY CONSORTIUM

Biospecimen

Retention: SAMPLES WITH DNA

Participating sites may collect biofluids, including DNA, plasma, serum, etc.

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Bulbo-Spinal Atrophy, X-Linked; Muscular Atrophy, Spinal; Bulbar Palsy, Progressive

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heredodegenerative Disorders, Nervous System; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Alexander Sherman

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Natalia Tarasenko

CONTACT

1617240346; ntarasenko@mgh.harvard.edu

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Center for Innovation and Bioinformatics

Study Record Dates

• First Submitted: June 15, 2023
• First Posted: July 28, 2023
• Study Start: April 1, 2015
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): December 31, 2030
• Last Updated: November 14, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: 10-30 days from the initial request's online submission
• Access Criteria: ALS/MND Natural History Consortium's Data Access committee reviews the merits of the requestors' research proposals and approves or denies data access

Locations

US (12); IL (2); IT (2)