Amyotrophic Lateral SclerosisPhase 1/2NADALS-001-ALSNCT07414212
Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Amyotrophic Lateral Sclerosis (ALS)
A Phase 1/2 study testing Acetylcysteine, EH301 and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Biogipuzkoa Health Research Institute at 1 site in Spain.
Trial summary
This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Acetylcysteine, EH301 and Riluzole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 1 siteSpain
- Study dates
- 25 Nov 2025to 25 Nov 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients diagnosed with ALS according to the Gold Coast criteria;
- Disease duration ≤ 18 months;
- Men and women aged 18 to 75 years;
- Total ALSFRS-R score ≥ 30 for all 12 categories;
- Forced vital capacity (FVC) ≥70%;
- The participant must receive treatment with Riluzole (50 mg twice daily) for ≥30 days prior to Day 1 (Visit 2) and is expected to remain on that dose until the final study visit.
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- Willingness and ability of the patient to comply with the requirements of the protocol during the study;
- Sign written informed consent prior to any study-related procedure;
- Acceptance by women to use at least one highly effective method of contraception during the study 30 days prior to taking the nutraceutical and investigational drug and throughout the study. The following are considered highly effective contraceptive methods:
- Combined hormonal methods (oral, patches, injectables, or implants).
- Hormonal or copper intrauterine devices (IUDs).
- Previous surgical sterilization (bilateral tubal ligation).
- Total sexual abstinence when consistent with the patient's usual lifestyle.
- Acceptance by men included in the study of the use of condoms in combination with an effective contraceptive method used by their female partner of childbearing age during treatment.
Who cannot join
- Presence of other neurodegenerative diseases;
- Significant cognitive impairment and/or dementia;
- Any psychiatric illness that could interfere with the study;
- Use of dietary supplements with high doses of vitamin B3 in the 30 days prior to inclusion in the study;
- Severe heart disease;
- Moderate to severe lung disease, such as emphysema, stage III-IV COPD;
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- Uncontrolled chronic asthma;
- Active cancer;
- Any metabolic, neoplastic, physically or mentally debilitating disease that could put the subject at risk or interfere with the study results;
- Genetically confirmed mitochondrial disease;
- Tracheostomized and/or gastrostomized patients;
- Participation in any clinical trial with an investigational product within 30 days or five half-lives of the previous agent, whichever is longer, prior to dosing;
- Any clinically significant laboratory abnormality that could directly affect compliance or safety;
- Allergy to NAC or any excipient, either in the investigational drug or in the EH301 nutraceutical;
- Patients with a short life expectancy in the investigator's judgment.
- [Women only] Pregnancy or breastfeeding for women of childbearing potential (i.e., <2 years postmenopausal or not surgically sterile);
- The participant is unwilling to use highly effective contraception during the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario Donostia
San Sebastián, Guipuzcoa, 20003
Patricia Garay Albizuri, +34 943006294, PATRICIA.GARAYALBIZURI@osakidetza.eus
Adolfo López de Munain
About this study
Official title: A Phase II, Single-center, Randomized Double Blind, Placebo Controlled Study to Evaluate the Effect of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Ambulant Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)
This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS.
The main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms.
Read more
Participants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring.
After completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Acetylcysteine, EH301, Riluzole
- Keywords
- Amyotrophic Lateral Sclerosis, Acetylcysteine, EH301, ALSFRS-R, ALS, NAC, Hospital Universitario Donostia, Biogipuzkoa, Adolfo López de Munain, NADALS-001-ALS-2021, Open Label Extension, NADALS
- 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.
AcetylcysteineDrug
Also called NAC
Daily dose of 600mg of effervescent powder
EH301Dietary supplement
Also called Nicotinamide riboside and pterostilbene
Daily dose of 1800mg of capsules
RiluzoleDrug
Daily dose of 100mg of tablets
| Study group | Role | What they receive |
|---|---|---|
| Acetylcysteine + EH301 | Experimental | Combination of 600 mg of NAC + 1800 mg of EH301 (1500 mg of NR + 300 mg of PTER) + 100 mg of Riluzole (standard treatment for both arms) Receives: Drug: Acetylcysteine; Dietary Supplement: EH301; Drug: Riluzole |
| Acetylcysteine Placebo + EH301 Placebo | Placebo comparator | Combination of NAC placebo + EH301 placebo + 100 mg of Riluzole (standard treatment for both arms) Receives: Drug: Riluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
ALSFRS-R scale
Change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) [range from 0 to 48, being 48 normal function and 0 complete function loss] to assess disease progression and evaluate the clinical efficacy and safety of the combination of NAC and EH301 during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Secondary outcomes (8)
Muscle strength
Muscle strength measured by hand dynamometry during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Ventilation-free survival
Percentage of participants who remain alive without requiring tracheotomy or permanent invasive ventilation during the randomized period (24 weeks) or, for those who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Quality of life (ALSAQ-40)
Quality of life assessed using the ALSAQ-40 questionnaire [range from 0 to 100, being 0 best health status and 100 worst health status] during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Respiratory function (%FVC)
Respiratory function measured as percent predicted forced vital capacity (%FVC) during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Time to gastrostomy
Time from baseline to placement of gastrostomy tube (percutaneous endoscopic gastrostomy or radiologically inserted gastrostomy). Participants in the open-label extension are followed for events up to 48 weeks.
Measured: Up to 48 weeks
Combined Assessment of Function and Survival (CAFS)
Combined Assessment of Function and Survival (CAFS) score calculated using ALSFRS-R change and survival status [no fixed numerical range; ranking score where higher values indicate better combined functional status and survival] during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Neurofilament light chain (NfL) biomarker
Neurofilament light chain (NfL) levels in serum and cerebrospinal fluid will be measured for biomarker assessment. Analyses will evaluate the change from baseline in NfL concentrations during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Serum lipidomic profile
Serum lipidomic components will be measured for biomarker assessment. Analyses will evaluate the change from baseline in the serum lipidomic profile during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Measured: Up to 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start25 Nov 2025
- Primary completion25 Nov 2026 (estimated)
- Study completion25 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biogipuzkoa Health Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07414212
- Sponsor's protocol ID
- NADALS-001-ALS-2021
- Other IDs
- 2024-519857-13-00
- Acronym
- NADALS-001-ALS
- Status last verified
- Feb 2026
- First submitted
- 2 Feb 2026
- Last updated
- 17 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Single-center, Randomized Double Blind, Placebo Controlled Study to Evaluate the Effect of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Ambulant Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT07414212
- Acronym / sponsor ID
- NADALS-001-ALS / NADALS-001-ALS-2021
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, Acetylcysteine, EH301, ALSFRS-R, ALS, NAC, Hospital Universitario Donostia, Biogipuzkoa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Biogipuzkoa Health Research InstituteUniversity, hospital or other
- Study start
- 25 Nov 2025 (actual)
- Primary completion
- 25 Nov 2026 (estimated)
- Study completion
- 25 Nov 2027 (estimated)
- First posted
- 17 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS.
The main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms.
Participants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring.
After completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Acetylcysteine + EH301 | Experimental | Drug: Acetylcysteine, Dietary Supplement: EH301, Drug: RiluzoleCombination of 600 mg of NAC + 1800 mg of EH301 (1500 mg of NR + 300 mg of PTER) + 100 mg of Riluzole (standard treatment for both arms) |
| Acetylcysteine Placebo + EH301 Placebo | Placebo comparator | Drug: RiluzoleCombination of NAC placebo + EH301 placebo + 100 mg of Riluzole (standard treatment for both arms) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Patients diagnosed with ALS according to the Gold Coast criteria;
- Disease duration ≤ 18 months;
- Men and women aged 18 to 75 years;
- Total ALSFRS-R score ≥ 30 for all 12 categories;
- Forced vital capacity (FVC) ≥70%;
Key exclusion criteria (17 listed)
- Presence of other neurodegenerative diseases;
- Significant cognitive impairment and/or dementia;
- Any psychiatric illness that could interfere with the study;
- Use of dietary supplements with high doses of vitamin B3 in the 30 days prior to inclusion in the study;
- Severe heart disease;
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 |
|---|---|---|
| AcetylcysteineAlso: NACDaily dose of 600mg of effervescent powder | Drug | Acetylcysteine + EH301 |
| EH301Also: Nicotinamide riboside and pterostilbeneDaily dose of 1800mg of capsules | Dietary supplement | Acetylcysteine + EH301 |
| RiluzoleDaily dose of 100mg of tablets | Drug | Acetylcysteine + EH301, Acetylcysteine Placebo + EH301 Placebo |
Primary outcome
ALSFRS-R scale
Time frame: Up to 48 weeks
Change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) [range from 0 to 48, being 48 normal function and 0 complete function loss] to assess disease progression and evaluate the clinical efficacy and safety of the combination of NAC and EH301 during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
8 secondary outcomes
Muscle strength
Time frame: Up to 48 weeks
Ventilation-free survival
Time frame: Up to 48 weeks
Quality of life (ALSAQ-40)
Time frame: Up to 48 weeks
Respiratory function (%FVC)
Time frame: Up to 48 weeks
Time to gastrostomy
Time frame: Up to 48 weeks
Combined Assessment of Function and Survival (CAFS)
Time frame: Up to 48 weeks
Neurofilament light chain (NfL) biomarker
Time frame: Up to 48 weeks
Serum lipidomic profile
Time frame: Up to 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
- CTISLink provided by the sponsor.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Acetylcysteine, EH301 and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Biogipuzkoa Health Research Institute at 1 site in Spain.
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 14 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lara Alameda Calvo (+34 943006140, lara.alamedacalvo@bio-gipuzkoa.eus), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALSFRS-R scale (time frame: Up to 48 weeks). 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 17 Feb 2026.