Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial (MICABO-ALS)

March 25, 2022 updated by: Dallas VA Medical Center
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that affects muscle function throughout the body. Weakness and muscle shrinkage begin either in the face, arm, or leg. A clinical ALS hallmark is rapidly progressive weight loss. Also, respiration is usually affected late in the disease, and death typically occurs as a consequence of respiratory failure. The median survival after disease onset is approximately 3-4 years.

While there are now two FDA-approved agents for patients with ALS, there are no interventions that have had a meaningful impact on the natural course of this disease. Riluzole prolongs survival by up to 12 weeks. Edaravone improves some aspects of neurological function in a small subset of patients, but that was ineffective in clinical studies that included ALS patients at all stages of disease.

Past failures to identify effective therapies reflect the complexity of ALS pathogenesis, in that no single therapeutic target has been identified. Thus, single agent or dual combination therapies are unlikely to succeed. Given the truly rapid and devastating nature of ALS and that there are no effective treatments for ALS, one can argue that it is critical to devise a different approach. Until the exact mechanisms that lead to ALS are identified, it is necessary to employ polytherapy which includes new agents that show promise.

This study will lay further groundwork on methodology for performing more definite trials in ALS. The study of secondary biomarkers in ALS is significant because there are currently no molecular or biochemical biomarkers for assessing therapeutic efficacy of drug treatments in ALS clinical trials. By doing this study, the study investigators hope to learn that high-dose 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. Participation in this research will last about 13 months

Study Type

Interventional

Enrollment (Anticipated)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75216
        • Recruiting
        • VA North Texas Health Care System
        • Contact:
          • Olaf Stuve, MD., Ph.D

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

19 years to 78 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criterion (Appendix 2).
  2. 21 to 80 years of age inclusive.
  3. If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.
  4. Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).

Exclusion Criteria:

  1. Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc.).
  2. Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc.) over the last 30 days.
  3. Infection with the human immunodeficiency virus (HIV)
  4. Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
  5. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Antioxidants
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
Over-the-counter anti-oxidants, namely vitamin E, NAc cysteine, L-cystine, Nicotinamide and Taurursodiol at defined doses

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measurement of serum NfL
Time Frame: 12 months
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measurement of functional decline in ALS
Time Frame: 12 months

Functional decline in ALS patients will be measured using the ALS Functional Rating Scale - Revised (ALSFRS-R), change will be measured from baseline to 12 months.

The ALSFRS-R is a quickly administered (five minutes) ordinal rating scale that assesses patients' capability and independence in 12 functional activities.
12 months
Frequency of serious adverse events and adverse events.
Time Frame: 12 months
The frequency of serious adverse events and adverse events will be summarized using frequency and percentages.
12 months
Survival analysis
Time Frame: 12 months
This is a robust measure of effect in this rapidly progressing terminal disease. Survival has been a standard outcome measure in past clinical trials.
12 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in serum CK level from baseline to 12 months
Time Frame: 12 months
CK levels will be measured in a clinical laboratory by colorimetric measurements. Analysis of serum CK levels will be conducted similar to serum NfL.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dallas VA Medical Center

Investigators

  • Principal Investigator: Olaf Stuve, M.D., Ph.D., Dallas VA Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

April 1, 2022

Primary Completion (Anticipated)

December 1, 2023

Study Completion (Anticipated)

December 1, 2023

Study Registration Dates

First Submitted

January 22, 2020

First Submitted That Met QC Criteria

January 24, 2020

First Posted (Actual)

January 28, 2020

Study Record Updates

Last Update Posted (Actual)

April 5, 2022

Last Update Submitted That Met QC Criteria

March 25, 2022

Last Verified

March 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 19-094

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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