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

NCT04562831 | Active Not Recruiting

• 1 other identifier: 98955
• Study Type: interventional
• Target: 380
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Change in vital capacity

  Change from baseline to 1 year

• Change in cognitive functions as assessed by the Edinburgh Cognitive Scale (ECAS)

  Change from baseline to 1 year

• Change of Neurofilament light chain (NFL) levels in serum

  Change from baseline to 1 year

• Change in quality of life as assessed by the quality of life questionnaire SF-36

  Change from baseline to 1 year

Other Outcomes (1)

• Overall survival

  Through study completion, 1 year

Study Arms (2)

Newly diagnosed ALS patients

EXPERIMENTAL

1. High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene) 2. Single dose EH301 (1000mg Nicotinamide riboside / 200mg Pterostilbene) 3. Placebo

Dietary Supplement: EH301 (Nicotinamide Riboside/Pterostilbene)

Earlier diagnosed ALS patients

EXPERIMENTAL

1. High dose EH301 (1500mg Nicotinamide riboside / 300mg Pterostilbene) 2. Placebo

Dietary Supplement: EH301 (Nicotinamide Riboside/Pterostilbene)

Interventions

EH301 (Nicotinamide Riboside/Pterostilbene) DIETARY_SUPPLEMENT

Comparison of 2 different dosages with placebo in newly diagnosed ALS patients and comparison of high dose with placebo in earlier ALS patients.

Earlier diagnosed ALS patients; Newly diagnosed ALS patients

Eligibility Criteria

• Age: 35 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Haukeland University Hospital: lead
• Elysium Health: collaborator

Study Sites (9)

Haukeland University Hospital

Bergen, Norway

Location

Vestre Viken HF

Drammen, Norway

Location

Helse Førde HF

Førde, Norway

Location

Helse Fonna HF

Haugesund, Norway

Location

Akershus University Hospital

Lørenskog, Norway

Location

Oslo University Hospital

Oslo, Norway

Location

Stavanger University Hospital

Stavanger, Norway

Location

Universitetssykehuset Nord-Norge

Tromsø, Norway

Location

St.Olavs Hospital HF

Trondheim, Norway

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

nicotinamide-beta-riboside; Pterocarpus marsupium

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Ole-Bjørn Tysnes

  Haukeland University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2020
• First Posted: September 24, 2020
• Study Start: October 7, 2020
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: January 12, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

NO (9)