Biomarkers in Different Types of Amyotrophic Lateral Sclerosis (ALS) Patients Being Treated With Edaravone

NCT04097158 | Recruiting DMC

• 1 other identifier: IRB#5190061
• Study Type: observational
• Target: 160
• Locations: 1 country
• Sites: 1

Brief Summary

This study is being conducted to help the investigators better understand how the new FDA approved medication Edaravone (also known as Radicava) works in subsets of patients with ALS. The investigators are also trying to understand if there are specific ALS patients, with different presentations of ALS, who might benefit most from this medication. Also, the investigators are following specific biomarkers to determine the optimal treatment duration in patients with different forms of ALS There is no study medication being offered in this trial. Edaravone is prescribed as part of regular care. In this trial we are collecting blood, urine, and spinal fluid samples in ALS patients who are taking Edaravone and ALS patients who are not taking Edaravone to measure certain markers that could indicate why the drug may be working in a specific type of ALS.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Define pharmacodynamic biomarkers of oxidative stress and antioxidant capacity in different ALS/MND phenotypes.

  The investigators aim to identify 4 cohorts of patients with distinct ALS/MND phenotypes and measure a panel of pharmacodynamic biomarkers of oxidative stress and antioxidant capacity in the CSF, blood, and urine.

  6 months

Study Arms (4)

Upper Motor Neuron predominant ALS

Other: Sample Collection

Lower Motor Neuron predominant ALS

Other: Sample Collection

Bulbar predominant ALS

Other: Sample Collection

Generalized ALS

Other: Sample Collection

Interventions

Sample Collection OTHER

The investigators will be collecting blood, urine, and spinal fluid samples.

Bulbar predominant ALS; Generalized ALS; Lower Motor Neuron predominant ALS; Upper Motor Neuron predominant ALS

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Anyone who meets the Inclusion/Exclusion Criteria

You may qualify if:

• Either possible, probable, or definite ALS, predominantly lower motor neuron disease, predominantly upper motor neuron disease, or bulbar
• With or without cognitive involvement
• Willing to participate
• On no experimental treatment
• Ages 18 - 85
• No prior exposure to Edaravone (Radicava)
• On a stable dose of Riluzole for 30 days or off Riluzole
• Male or female
• Females of childbearing age must use contraception

You may not qualify if:

• Unstable medical illness
• Abnormal liver function (\>2x ULN)
• Unlikely to survive for 26 weeks

Sponsors & Collaborators

• Loma Linda University: lead
• Temple University: collaborator
• Thomas Jefferson University: collaborator
• University of Southern California: collaborator
• Northwestern University: collaborator

Study Sites (1)

Loma Linda University

Loma Linda, California, 92354, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Blood Spinal Fluid Urine

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Specimen Handling

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

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• Jeffrey Rosenfeld, PhD, MD

  Loma Linda University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Imran Qasim

CONTACT

(909) 558-2037; sqasim@llu.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2019
• First Posted: September 20, 2019
• Study Start: October 8, 2019
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: May 21, 2025

Record last verified: 2025-05

Locations

US (1)