Personalized Treatments for ALS

No longer recruiting at 1 trial location
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Overseen ByRichard Bedlack, MD, PhD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method to slow the progression of ALS, a disease affecting nerve cells in the brain and spinal cord, leading to muscle control loss. Participants will be grouped based on blood work into one of four ALS-related categories: neuroinflammation (inflammation in the brain and spinal cord), oxidative stress (damage from "free radicals"), impaired autophagy (difficulty clearing damaged cells), or mitochondrial dysfunction (issues with energy production). Each group will receive a specific supplement—Astaxanthin, Protandim, Melatonin, or MitoQ (targeting mitochondrial dysfunction)—designed to address their specific issue. The study seeks individuals diagnosed with ALS who can swallow tablets. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in ALS treatment.

Do I need to stop taking my current medications for the ALS trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot be currently taking any of the four investigational treatments used in this trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that the four supplements tested in this ALS trial have varying levels of safety information.

Astaxanthin hasn't been specifically tested in people with ALS, but it is generally considered safe and affordable. Melatonin appears safe for ALS patients, though the optimal dose remains unknown. Some studies suggest it might slow disease progression.

MitoQ has shown positive results in animal studies of ALS and appears fairly safe. It targets mitochondria, which help produce energy in cells.

Protandim has been studied for its impact on reducing cell damage. Clinical studies found no major side effects, but its benefits for ALS remain unclear.

Overall, these treatments are usually well-tolerated, but more research is needed to understand their specific effects on ALS.12345

Why are researchers excited about this trial's treatments for ALS?

Researchers are excited about these treatments for ALS because they target specific underlying mechanisms linked to the disease. Unlike traditional treatments like riluzole and edaravone that primarily aim to slow disease progression, these investigational treatments focus on different aspects of cellular health. Astaxanthin, melatonin, MitoQ, and Protandim are believed to combat oxidative stress, reduce neuroinflammation, support mitochondrial function, and enhance autophagy, respectively. This approach is unique because it aims to address the root causes of motor neuron damage, offering a potentially more comprehensive strategy for managing ALS.

What evidence suggests that this trial's treatments could be effective for ALS?

Research suggests that astaxanthin, one of the treatments in this trial, may help slow ALS by protecting nerve cells. Participants may also receive melatonin, which studies have linked to slower disease progression and longer survival in ALS patients. MitoQ, another treatment option, showed promise in animal studies by improving cellular energy production, potentially slowing ALS. Protandim, also under study, activates a protective process in cells and has been linked to improved ALS symptoms and some survival benefits in certain studies. Each treatment targets a specific aspect of ALS, aiming to slow the disease's progress.12467

Are You a Good Fit for This Trial?

This trial is for individuals with Motor Neuron Disease, specifically Amyotrophic Lateral Sclerosis (ALS). Participants will be categorized based on their blood work into groups reflecting different aspects of ALS pathology and receive personalized supplements accordingly.

Inclusion Criteria

Patient is able to understand and express informed consent (in the opinion of the site investigator)
I have been diagnosed with ALS.
Patient is able to read and write English
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Exclusion Criteria

Patient has a medical or psychiatric illness that could in the investigator's opinion interfere with the patient's ability to participate in this study
Currently or recently (within 30 days) taking any of the 4 investigational treatments being used in this trial
Pregnant women or women currently breastfeeding
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Baseline Assessment

Participants provide demographics, disease characteristics, co-morbidities, concomitant medications, and baseline ALSFRS-R score. Blood is drawn for DIGAP classification and baseline biomarkers.

1 week
1 visit (in-person)

Initial Monitoring

Participants are monitored monthly via phone for adverse events, new co-morbidities, and concomitant medications. ALSFRS-R scores are updated.

3 months
3 visits (virtual)

Treatment

Participants receive one of four individualized supplements based on DIGAP classification for 6 months. Blood draws for repeat biomarkers occur at months 3, 5, and 9.

6 months
3 visits (in-person), 3 visits (virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion.

1 month
1 visit (virtual)

What Are the Treatments Tested in This Trial?

Interventions

  • Astaxanthin
  • Melatonin
  • MitoQ
  • Protandim
Trial Overview The trial tests a platform that categorizes ALS patients into four disease categories. Depending on the category—neuroinflammation, oxidative stress, impaired autophagy & axonal transport, or mitochondrial dysfunction—participants get Astaxanthin, Protandim, Melatonin, or MitoQ respectively for 6 months.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Group I: Oxidative StressExperimental Treatment1 Intervention
Group II: NeuroinflammationExperimental Treatment1 Intervention
Group III: Mitochondrial DysfunctionExperimental Treatment1 Intervention
Group IV: Impaired Autophagy and Axonal TransportExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Duke University

Lead Sponsor

Trials
2,495
Recruited
5,912,000+

Temple University

Collaborator

Trials
321
Recruited
89,100+

Citations

Amyotrophic Lateral Sclerosis: Insights and New Prospects in ...These drugs marginally slow down the progression of the disease but do not cure it. Many novel therapeutics are entering clinical trials with ...
Full article: ALSUntangled # 69: astaxanthinHere we review astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects.
Biological and neurological activities of astaxanthin (Review)Astaxanthin has received attention for its protective effects against neurological disorders, including Alzheimer's disease, Parkinson's disease, amyotrophic ...
4.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/36694292/
ALSUntangled # 69: astaxanthinHere we review astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects.
ALSUntangled # 69: astaxanthin. Amyotroph Lateral Scler ...Here we review astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects.
Antioxidant Alternatives in the Treatment of Amyotrophic ...Investigation of the safety and efficacy of edaravone in 20 ALS patients who received this antioxidant intravenously indicated that this drug is safe and may ...
ALSUntangled # 69: astaxanthin. - Scholars@Duke publicationThere have been no trials of astaxanthin in people living with ALS. Natural astaxanthin appears to be safe and inexpensive. Based on the above information, we ...
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