Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial

NCT04244630 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 19-094
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research is to investigate the validity of a previous clinical trial named EH301, which showed beneficial effects of anti-oxidant therapies in patients with amyotrophic lateral sclerosis (ALS). If validated by this study, providing over-the-counter anti-oxidants would be a simple, low risk, low-cost approach to significantly slow or stop the progression of ALS, for which currently no effective treatment exists. It is currently thought that oxidative stress is a major cause of ALS. The study investigators are therefore planning to expand the original scope of the previous trial by including anti-oxidants at high doses that were not previously used. All of these compounds are considered safe.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (1)

• Measurement of serum NfL

  The change in log serum NfL level from baseline to 12 months will be assessed using a linear mixed model and the differences in NfL level at 12 months from baseline assessed using a paired t-test.

  12 months

Secondary Outcomes (3)

• Measurement of functional decline in ALS

  12 months

• Frequency of serious adverse events and adverse events.

  12 months

• Survival analysis

  12 months

Other Outcomes (1)

• Change in serum CK level from baseline to 12 months

  12 months

Study Arms (1)

Antioxidants

OTHER

Eligible patients will receive over-the-counter anti-oxidants, namely vitamin E, NAc cysteine, L-cystine, Nicotinamide and Taurursodiol at defined doses.

Combination Product: Antioxidants

Interventions

Antioxidants COMBINATION_PRODUCT

Over-the-counter anti-oxidants, namely vitamin E, NAc cysteine, L-cystine, Nicotinamide and Taurursodiol at defined doses

Antioxidants

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criterion (Appendix 2).
• to 80 years of age inclusive.
• If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.
• Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).

You may not qualify if:

• Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc.).
• Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc.) over the last 30 days.
• Infection with the human immunodeficiency virus (HIV)
• Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
• History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols. 6 Receipt of any investigational drug within the past 30 days.

Sponsors & Collaborators

• Dallas VA Medical Center: lead

Study Sites (1)

VA North Texas Health Care System

Dallas, Texas, 75216, United States

RECRUITING

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MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Antioxidants

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)

Biological Factors; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Protective Agents; Physiological Effects of Drugs; Specialty Uses of Chemicals

Study Officials

• Olaf Stuve, M.D., Ph.D.

  Dallas VA Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rehana Hussain, M.Sc.

CONTACT

214-648-7244; rehana.hussain@utsouthwestern.edu

Shirley OLeary, NP

CONTACT

214-857-4459; Shirley.OLeary@va.gov

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: FED
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2020
• First Posted: January 28, 2020
• Study Start: November 2, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: August 24, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)