Clinical trial NCT04562831: The NO-ALS Study: a Trial of Nicotinamide/pterostilbene Supplement in ALS.
Official title: A Randomized Placebo-controlled Trial of Nicotinamide/pterostilbene Supplement in ALS: the NO-ALS Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Haukeland University Hospital
Sponsor class: OTHER
Collaborators: name: Elysium Health
First posted: 2020-09-24
Enrollment: 380
Sex: ALL
Minimum age (years): 35
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
timeFrame: Change from baseline to 1 year
Secondary outcomes:
measure: Change in vital capacity
timeFrame: Change from baseline to 1 year
measure: Change in cognitive functions as assessed by the Edinburgh Cognitive Scale (ECAS)
timeFrame: Change from baseline to 1 year
measure: Change of Neurofilament light chain (NFL) levels in serum
timeFrame: Change from baseline to 1 year
measure: Change in quality of life as assessed by the quality of life questionnaire SF-36
timeFrame: Change from baseline to 1 year
Other outcomes:
measure: Overall survival
timeFrame: Through study completion, 1 year
Locations:
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Drammen
country: Norway
facility: Vestre Viken HF
city: Førde
country: Norway
facility: Helse Førde HF
city: Haugesund
country: Norway
facility: Helse Fonna HF
city: Lørenskog
country: Norway
facility: Akershus University Hospital
city: Oslo
country: Norway
facility: Oslo University Hospital
city: Stavanger
country: Norway
facility: Stavanger University Hospital
city: Tromsø
country: Norway
facility: Universitetssykehuset Nord-Norge
city: Trondheim
country: Norway
facility: St.Olavs Hospital HF
Study officials:
affiliation: Haukeland University Hospital
name: Ole-Bjørn Tysnes
role: PRINCIPAL_INVESTIGATOR
References: