The NO-ALS Study: A Trial of Nicotinamide/Pterostilbene Supplement in ALS.

A Randomized Placebo-controlled Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Study

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is 3 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK per year

Research i.a. from the investigators department has shown that increased activity in histone deacetylation enzymes (sirtuins) together with increased access to NAD can delay disease progression. Nicotinamide riboside (NR) can increase cells' access to NAD and Pterostilben will stimulate sirtuins.

The investigators want to study whether combination therapy with NR and Pterostilben can inhibit neurodegeneration in ALS and thereby delay disease development, increase survival and improve quality of life in ALS.

In the study, the investigators will use 2 different dosages on the active treatment and strength calculations show that 180 patients are needed to show a rather weak effect. Patients will be recruited in collaboration with hospitals in Helse Vest, AHUS, Drammen, OUS and St. Olavs hospital.

Study Overview

• Status: Active, not recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Dietary supplement: EH301 (Nicotinamide Riboside/Pterostilbene)

• Study Type: Interventional
• Enrollment (Estimated): 380
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Norway
  • Bergen, Norway
    • Haukeland University Hospital
  • Drammen, Norway
    • Vestre Viken HF
  • Førde, Norway
    • Helse Førde HF
  • Haugesund, Norway
    • Helse Fonna HF
  • Lørenskog, Norway
    • Akershus University Hospital
  • Oslo, Norway
    • Oslo University Hospital
  • Stavanger, Norway
    • Stavanger University Hospital
  • Tromsø, Norway
    • Universitetssykehuset Nord-Norge
  • Trondheim, Norway
    • St.Olavs Hospital HF

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 35 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Arm 1 (newly diagnosed ALS patients)

• Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.
• MR of the brain and cervical spine cannot explain symptoms.
• Diagnosed with likely ALS within 6 months from enrolment and treated with Riluzole 50mg x 2
• Symptom onset no longer than 2 year prior to inclusion.
• ALS-FRC-R of 36 or more (not any item below 2).
• Age equal to or greater than 35 years at time of enrollment

Arm 2 (earlier diagnosed ALS patients)

• Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.
• MR of the brain and cervical spine cannot explain symptoms.
• Treated with Riluzole 50mg x 2.

Exclusion Criteria:

• Dementia, FTD or other neurodegenerative disorder at baseline visit
• Any psychiatric disorder that would interfere with compliance in the study.
• Use of high dose vitamin B3 supplementation within 30 days of enrollment
• Metabolic, neoplastic, or other physically or mentally debilitating disorder at baseline visit.
• Genetically confirmed mitochondrial disease
• Patients who become tracheostomized as part of the treatment of ALS
• Patients with short expected survival at the discretion of the investigator. Such cases cannot be expected to follow protocol procedures.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Newly diagnosed ALS patients; High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene); Single dose EH301 (1000mg Nicotinamide riboside / 200mg Pterostilbene); Placebo	Dietary supplement: EH301 (Nicotinamide Riboside/Pterostilbene); Comparison of 2 different dosages with placebo in newly diagnosed ALS patients and comparison of high dose with placebo in earlier ALS patients.
Experimental: Earlier diagnosed ALS patients; High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene); Placebo	Dietary supplement: EH301 (Nicotinamide Riboside/Pterostilbene); Comparison of 2 different dosages with placebo in newly diagnosed ALS patients and comparison of high dose with placebo in earlier ALS patients.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)	ALSFRS-R is a validated rating instrument for monitoring the progression of disability in patients with ALS. The minimum score is 0 and the maximum score is 48, the higher score the more function is retained.	Change from baseline to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in vital capacity	Vital capacity in sitting position and supine by spirometry. Spirometry measures the amount of air that can be inhaled or exhaled and vital capacity is the volume of air breathed out after the deepest inhalation, the higher value the better vital capacity.	Change from baseline to 1 year
Change in cognitive functions as assessed by the Edinburgh Cognitive Scale (ECAS)	ECAS determines cognitive and behavioural changes of patients suffering from ALS. The minimum score is 0 and the maximum score is 136, the lower the score the greater the deficit.	Change from baseline to 1 year
Change of Neurofilament light chain (NFL) levels in serum	NFL levels in serum, baseline values and changes during the study.	Change from baseline to 1 year
Change in quality of life as assessed by the quality of life questionnaire SF-36	SF-36 is a 36-item patient-reported survey of patient health. The minimum score is 0 and the maximum score is 100. The higher the score the less disability.	Change from baseline to 1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	Survival of patients through the study	Through study completion, 1 year

Collaborators and Investigators

• Sponsor: Haukeland University Hospital
• Collaborators: Elysium Health
• Investigators: Principal Investigator: Ole-Bjørn Tysnes, Haukeland University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): October 7, 2020
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2026

Study Registration Dates

• First Submitted: September 10, 2020
• First Submitted That Met QC Criteria: September 18, 2020
• First Posted (Actual): September 24, 2020

Study Record Updates

• Last Update Posted (Estimated): January 12, 2026
• Last Update Submitted That Met QC Criteria: January 9, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Antimetabolites; Micronutrients; Hypolipidemic Agents; Lipid Regulating Agents; Vitamins; Vitamin B Complex; nicotinamide-beta-riboside; Pterocarpus marsupium
• Other Study ID Numbers: 98955

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No