Personalized Treatments for ALS

Recruiting at 1 trial location

Overseen By Hailey Zampa

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Duke University

Must not be taking: Astaxanthin, Protandim, Melatonin, MitoQ

Disqualifiers: Pregnancy, Breastfeeding, Psychiatric illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 9

Duration: 6 months

50 Participants Needed

Trial Summary

What is the purpose of this trial?

GenieUs developed an analysis platform that will be tested to separate study participants with ALS into four categories based on blood work. These general categories are neuroinflammation, oxidative stress, impaired autophagy \& axonal transport, and mitochondrial dysfunction. Once a disease category is established, participants in this study will receive one of four individualized supplements for 6 months and we will determine whether these are slowing ALS progression: Astaxanthin will be given for the category of neuroinflammation, Protandim for oxidative stress, Melatonin for impaired autophagy and MitoQ for mitochondrial dysfunction. During the first 3 months, participants will have routine monitoring and in months 3 through 9 they will receive the assigned supplement.

Eligibility Criteria

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

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

Timeline

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)

Treatment Details

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.

Participant Groups

Group I: Oxidative Stress Experimental Treatment 1 Intervention

Study participants in this category are expected to have too many damaging "free radical" chemicals in their brains and spinal cords.

Group II: Neuroinflammation Experimental Treatment 1 Intervention

Study participants in this category are expected to have inflammation in their brains and spinal cords.

Group III: Mitochondrial Dysfunction Experimental Treatment 1 Intervention

Study participants in this category are expected to have motor neurons that are unable to produce normal amounts of energy.

Group IV: Impaired Autophagy and Axonal Transport Experimental Treatment 1 Intervention

Study participants in this category are expected to have motor neurons that have trouble transporting materials up and down their length, and/or trouble with the turnover of damaged proteins and intracellular structures.

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+