Amyotrophic Lateral Sclerosis ALSPhase 2NCT06429059
ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments
A Phase 2 study testing Astaxanthin, Protandim and Melatonin in people with Amyotrophic Lateral Sclerosis ALS, run by Duke University at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis ALS
- What is being tested?
- Astaxanthin, Protandim and Melatonin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 2 sitesUnited States
- Study dates
- 12 Jun 2024to 24 Jun 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged at least 18 years.
- Sporadic or familial ALS diagnosed as per Gold Coast Criteria.
- Patient is able to understand and express informed consent (in the opinion of the site investigator).
- Patient is able to read and write English.
- Patient is expected to survive for the duration of the trial.
- Able to swallow tablets at enrollment and expected to be able to swallow tablets for the duration of the trial.
- Women must not be pregnant (will have evidence of a negative pregnancy test obtained by study team at baseline, or by local physician within past 7 days or be post-menopausal)
- Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception, or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.
Who cannot join
- Actively or recently (within past 30 days) participating in another intervention trial.
- Currently or recently (within 30 days) taking any of the 4 investigational treatments being used in this trial.
- Prior side effects from any of the 4 investigational treatments being used in this trial.
- 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.
- Pregnant women or women currently breastfeeding.
- Life expectancy shorter than the duration of the trial.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Duke University Medical Center
Durham, North Carolina, 27705
Temple University
Philadelphia, Pennsylvania, 19122
About this study
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.
Read the detailed description
This will be a widely inclusive, largely remote/virtual, two-center, open-label pilot trial utilizing 50 participants as their own controls. Following informed consent and screening, participants will provide demographics, disease characteristics, co-morbidities, and concomitant medications. They will have a baseline ALSFRS-R score obtained and blood will be drawn for DIGAP classification, PBMCs (which will be used to generate iPSCs from which motor neurons and/or microglia can be generated), baseline mechanistic biomarkers and baseline neurofilament light chain. A urine pregnancy test will be obtained for pre-menopausal females who have not had one by their own doctor in the past 7 days. Each month after that, they will be contacted by phone by study coordinators to review adverse events, new co-morbidities, and concomitant medications, and to generate a new ALSFRS-R score. At month 3, DIGAP classification will be revealed to each participant and based on this, they will receive 1 of 4 treatments. They will take their assigned treatment for 6 months. At months 3, 5 and 9 they will be asked to return for in person blood draws for repeat mechanistic biomarkers and neurofilament light chain measurements. All of the described blood tests and investigational treatments are being performed exclusively for research purposes.
- Conditions
- Amyotrophic Lateral Sclerosis ALS
- Being tested
- Astaxanthin, Protandim, Melatonin, MitoQ
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AstaxanthinDrug
Astaxanthin is a red-orange natural pigment belonging to a group of carotenoids called xanthophylls. In nature, astaxanthin is synthesized by microalgae and phytoplankton and biomagnifies in higher marine animals through the food chain. Natural astaxanthin is available as capsules, soft gels, tablets, powders, biomass, creams, energy drinks, oils and extracts and often contains other carotenoids. The compound is available as a United States Pharmacopeia (USP) verified supplement which ensures federally recognized standards for quality and purity (https://www.quality-supplements.org/verified-products/verified-products-listings).
ProtandimDrug
Protandim is an oral tablet derived from five different plants: Silybum marianum (milk thistle), Withania somnifera (Ashwagandha), Camellia sinensis (green tea), Curcuma longa (turmeric) and Bacopa monniera.
MelatoninDrug
Melatonin is a hormone that has long been known to play a role in regulating sleep. Melatonin supplements are commonly used to treat insomnia, but in recent years, melatonin has been found to play a wider role in human physiology including the potential regulation of autophagy.
MitoQDrug
The active ingredient in MitoQ is ubiquinone, the same as found in coenzyme Q10 and idebenone. However, the ubiquinone in MitoQ is attached to a positively charged, lipophilic molecule called TPP (triphenyl phosphonium), which allows it to selectively accumulate in mitochondria. This makes it more potent than untargeted ubiquinone analogs at protecting mitochondria in cultured cells. It can be administered orally and, at least in animals, can cross the blood brain barrier and accumulate in brain mitochondria.
| Study group | Role | What they receive |
|---|---|---|
| Neuroinflammation | Experimental | Study participants in this category are expected to have inflammation in their brains and spinal cords. Receives: Drug: Astaxanthin |
| Oxidative Stress | Experimental | Study participants in this category are expected to have too many damaging "free radical" chemicals in their brains and spinal cords. Receives: Drug: Protandim |
| Impaired Autophagy and Axonal Transport | Experimental | 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. Receives: Drug: Melatonin |
| Mitochondrial Dysfunction | Experimental | Study participants in this category are expected to have motor neurons that are unable to produce normal amounts of energy. Receives: Drug: MitoQ |
What is being measured
The primary outcome is the main question the study is designed to answer.
ALS Functional Rating Scale, Revised (ALSFRS-R)
A quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. The total score ranges from 0 (maximum disability) to 48 (normal function). A higher score indicates greater function.
Measured: Baseline, month 3, month 5, and month 9
Secondary outcomes (6)
Neurofilament Light Chain Levels
Neurofilament light chain levels are elevated in the spinal fluid and the blood of patients with ALS and other neurodegenerative diseases, and higher levels predict more severe disease progression. These levels rise dramatically when asymptomatic carriers of ALS-causing genetic mutations begin to convert to an ALS phenotype.
Measured: Baseline, month 3, month 5 (2 months into treatment) and month 9 (6 months into treatment)
Neuroinflammation Measured by C-reactive Protein (CRP)
Used to measure neuroinflammation. CRPs are known to be abnormal in patients with ALS, their levels correlate with disease progression and they can be altered by drugs in trials.
Measured: Baseline, 3 months, 5 months and 9 months
Neuroinflammation Measured by Monocyte Chemoattractant Protein-1 (MCP-1), Also Known as CCL2
Used to measure neuroinflammation. CCL2s are known to be abnormal in patients with ALS, their levels correlate with disease progression and they can be altered by drugs in trials.
Measured: Baseline, 3 months, 5 months and 9 months
Oxidative Stress Measured by Total Antioxidant Capacity (TAC)
Used to measure oxidative stress.
Measured: Baseline, 3 months, 5 months, and 9 months
Oxidative Stress Measured by Uric Acid Levels.
Uric acid is an antioxidant and levels of uric acid have been reported to be lower in ALS subjects and correlated with progression. It has also been shown to be responsive to treatments in trials
Measured: Baseline, 3 months, 5 months, and 9 months
Mitochondrial Dysfunction Measured by Lactate
Compromised mitochondrial oxidative phosphorylation shifts the cellular bioenergetic system to anaerobic respiration and increases the level of lactate. Lactate has been used as a biomarker for mitochondrial disease in many previous studies.
Measured: Baseline, 3 months, 5 months, and 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2024
- Study start12 Jun 2024
- Primary completion22 May 2025
- Study completion24 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral Sclerosis ALSStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- At month 3, DIGAP classification will be revealed to each participant and based on this, they will receive 1 of 4 treatments. They will take their assigned treatment for 6 months.
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- Temple University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06429059
- Sponsor's protocol ID
- Pro00114385
- Status last verified
- Oct 2026
- First submitted
- 9 Apr 2024
- Last updated
- 27 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments
- ClinicalTrials.gov ID
- NCT06429059
- Acronym / sponsor ID
- Pro00114385
- Conditions
- Amyotrophic Lateral Sclerosis ALS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- Temple University
- Study start
- 12 Jun 2024 (actual)
- Primary completion
- 22 May 2025 (actual)
- Study completion
- 24 Jun 2025 (actual)
- First posted
- 24 May 2024 (actual)
- Results
- Posted 27 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This will be a widely inclusive, largely remote/virtual, two-center, open-label pilot trial utilizing 50 participants as their own controls. Following informed consent and screening, participants will provide demographics, disease characteristics, co-morbidities, and concomitant medications. They will have a baseline ALSFRS-R score obtained and blood will be drawn for DIGAP classification, PBMCs (which will be used to generate iPSCs from which motor neurons and/or microglia can be generated), baseline mechanistic biomarkers and baseline neurofilament light chain. A urine pregnancy test will be obtained for pre-menopausal females who have not had one by their own doctor in the past 7 days. Each month after that, they will be contacted by phone by study coordinators to review adverse events, new co-morbidities, and concomitant medications, and to generate a new ALSFRS-R score. At month 3, DIGAP classification will be revealed to each participant and based on this, they will receive 1 of 4 treatments. They will take their assigned treatment for 6 months. At months 3, 5 and 9 they will be asked to return for in person blood draws for repeat mechanistic biomarkers and neurofilament light chain measurements. All of the described blood tests and investigational treatments are being performed exclusively for research purposes.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- At month 3, DIGAP classification will be revealed to each participant and based on this, they will receive 1 of 4 treatments. They will take their assigned treatment for 6 months.
| Arm | Type | Receives |
|---|---|---|
| Neuroinflammation | Experimental | Drug: AstaxanthinStudy participants in this category are expected to have inflammation in their brains and spinal cords. |
| Oxidative Stress | Experimental | Drug: ProtandimStudy participants in this category are expected to have too many damaging "free radical" chemicals in their brains and spinal cords. |
| Impaired Autophagy and Axonal Transport | Experimental | Drug: MelatoninStudy 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. |
| Mitochondrial Dysfunction | Experimental | Drug: MitoQStudy participants in this category are expected to have motor neurons that are unable to produce normal amounts of energy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female aged at least 18 years.
- Sporadic or familial ALS diagnosed as per Gold Coast Criteria.
- Patient is able to understand and express informed consent (in the opinion of the site investigator).
- Patient is able to read and write English.
- Patient is expected to survive for the duration of the trial.
Key exclusion criteria (6 listed)
- Actively or recently (within past 30 days) participating in another intervention trial.
- Currently or recently (within 30 days) taking any of the 4 investigational treatments being used in this trial.
- Prior side effects from any of the 4 investigational treatments being used in this trial.
- 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.
- Pregnant women or women currently breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AstaxanthinAstaxanthin is a red-orange natural pigment belonging to a group of carotenoids called xanthophylls. In nature, astaxanthin is synthesized by microalgae and phytoplankton and biomagnifies in higher marine animals through the food chain. Natural astaxanthin is available as capsules, soft gels, tablets, powders, biomass, creams, energy drinks, oils and extracts and often contains other carotenoids. The compound is available as a United States Pharmacopeia (USP) verified supplement which ensures federally recognized standards for quality and purity (https://www.quality-supplements.org/verified-products/verified-products-listings). | Drug | Neuroinflammation |
| ProtandimProtandim is an oral tablet derived from five different plants: Silybum marianum (milk thistle), Withania somnifera (Ashwagandha), Camellia sinensis (green tea), Curcuma longa (turmeric) and Bacopa monniera. | Drug | Oxidative Stress |
| MelatoninMelatonin is a hormone that has long been known to play a role in regulating sleep. Melatonin supplements are commonly used to treat insomnia, but in recent years, melatonin has been found to play a wider role in human physiology including the potential regulation of autophagy. | Drug | Impaired Autophagy and Axonal Transport |
| MitoQThe active ingredient in MitoQ is ubiquinone, the same as found in coenzyme Q10 and idebenone. However, the ubiquinone in MitoQ is attached to a positively charged, lipophilic molecule called TPP (triphenyl phosphonium), which allows it to selectively accumulate in mitochondria. This makes it more potent than untargeted ubiquinone analogs at protecting mitochondria in cultured cells. It can be administered orally and, at least in animals, can cross the blood brain barrier and accumulate in brain mitochondria. | Drug | Mitochondrial Dysfunction |
Primary outcome
ALS Functional Rating Scale, Revised (ALSFRS-R)
Time frame: Baseline, month 3, month 5, and month 9
A quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. The total score ranges from 0 (maximum disability) to 48 (normal function). A higher score indicates greater function.
6 secondary outcomes
Neurofilament Light Chain Levels
Time frame: Baseline, month 3, month 5 (2 months into treatment) and month 9 (6 months into treatment)
Neuroinflammation Measured by C-reactive Protein (CRP)
Time frame: Baseline, 3 months, 5 months and 9 months
Neuroinflammation Measured by Monocyte Chemoattractant Protein-1 (MCP-1), Also Known as CCL2
Time frame: Baseline, 3 months, 5 months and 9 months
Oxidative Stress Measured by Total Antioxidant Capacity (TAC)
Time frame: Baseline, 3 months, 5 months, and 9 months
Oxidative Stress Measured by Uric Acid Levels.
Time frame: Baseline, 3 months, 5 months, and 9 months
Mitochondrial Dysfunction Measured by Lactate
Time frame: Baseline, 3 months, 5 months, and 9 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Astaxanthin, Protandim and Melatonin in people with Amyotrophic Lateral Sclerosis ALS, run by Duke University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALS Functional Rating Scale, Revised (ALSFRS-R) (time frame: Baseline, month 3, month 5, and month 9). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.