Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial (MICABO-ALS)
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|ClinicalTrials.gov Identifier: NCT04244630|
Recruitment Status : Recruiting
First Posted : January 28, 2020
Last Update Posted : April 5, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis (ALS)||Combination Product: Antioxidants||Phase 2|
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 (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial|
|Estimated Study Start Date :||April 1, 2022|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2023|
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
- 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.
- 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.
- 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.
- 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||21 Years to 80 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criterion (Appendix 2).
- 21 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).
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04244630
|Contact: Rehana Hussain, M.Sc.||firstname.lastname@example.org|
|Contact: Shirley OLeary, NP||214-857-4459||Shirley.OLeary@va.gov|
|United States, Texas|
|VA North Texas Health Care System||Recruiting|
|Dallas, Texas, United States, 75216|
|Contact: Olaf Stuve, MD., Ph.D|
|Principal Investigator:||Olaf Stuve, M.D., Ph.D.||Dallas VA Medical Center|
|Responsible Party:||Dallas VA Medical Center|
|Other Study ID Numbers:||
|First Posted:||January 28, 2020 Key Record Dates|
|Last Update Posted:||April 5, 2022|
|Last Verified:||March 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|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|
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs