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

Oxidative Markers and Efficacy in Amyotrophic Lateral Sclerosis (ALS) Phenotypes Treated With Edaravone

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.

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Other: Sample Collection

• Study Type: Observational
• Enrollment (Estimated): 160

Contacts and Locations

• Study Contact: Name: Imran Qasim; Phone Number: 25388 (909) 558-2037; Email: sqasim@llu.edu

Study Locations

• United States
  • California
    • Loma Linda, California, United States, 92354
      • Recruiting
      • Loma Linda University
      • Contact: Imran Qasim; Phone Number: 25388 909-558-2037; Email: sqasim@llu.edu
      • Principal Investigator: Jeffrey Rosenfeld, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Anyone who meets the Inclusion/Exclusion Criteria

Description

Inclusion Criteria:

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

Exclusion Criteria:

1. Unstable medical illness
2. Abnormal liver function (>2x ULN)
3. Unlikely to survive for 26 weeks

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Upper Motor Neuron predominant ALS	Other: Sample Collection; The investigators will be collecting blood, urine, and spinal fluid samples.
Lower Motor Neuron predominant ALS	Other: Sample Collection; The investigators will be collecting blood, urine, and spinal fluid samples.
Bulbar predominant ALS	Other: Sample Collection; The investigators will be collecting blood, urine, and spinal fluid samples.
Generalized ALS	Other: Sample Collection; The investigators will be collecting blood, urine, and spinal fluid samples.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Loma Linda University
• Collaborators: Northwestern University; University of Southern California; Temple University; Thomas Jefferson University
• Investigators: Principal Investigator: Jeffrey Rosenfeld, PhD, MD, Loma Linda University

Study record dates

Study Major Dates

• Study Start (Actual): October 8, 2019
• Primary Completion (Estimated): September 1, 2024
• Study Completion (Estimated): September 1, 2024

Study Registration Dates

• First Submitted: September 18, 2019
• First Submitted That Met QC Criteria: September 18, 2019
• First Posted (Actual): September 20, 2019

Study Record Updates

• Last Update Posted (Estimated): December 19, 2023
• Last Update Submitted That Met QC Criteria: December 18, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: IRB#5190061