Clinical trial NCT05095571: A Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Extension Study
Official title: A Randomized Placebo-controlled Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Study. Extension Protocol
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: 2021-10-27
Enrollment: 300
Sex: ALL
Minimum age (years): 35
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: The study is a multicenter open label study as a follow up of the NO-ALS trial. Newly diagnosed ALS cases were included in the study arm 1. Earlier ALS cases were included in study arm 2 to allow patients with earlier ALS to participate in the study as the treatment options outside the study were limited. The NO-ALS study will be stopped when we reach 180 patients in the group with newly diagnosed ALS cases (arm 1). This is the follow up study for patients completing 1 year follow up in the NO-ALS study.
Primary purpose: TREATMENT

Brief summary: Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The mean survival from the time of diagnosis is 2.5 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK (Norwegian kroner) per year.

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

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

In the NO-ALS extension study the investigators will follow the patients who completed the original NO-ALS study. Objectives are to evaluate adverse events and give patients possibility of compassionate use, and secondarily to see if the combination of NR and pterostilbene (EH301) will decrease progression of motor symptoms and loss of vital capacity, and increase survival time in patients with ALS.


Eligibility criteria: Inclusion Criteria:

* Patients who fulfilled the criteria for the NO-ALS study and have completed the study will be proposed inclusion in the NO-ALS extension study protocol. Patients from both arm 1 and arm 2 in the NO-ALS study will be allowed inclusion in the prolongation study

Exclusion Criteria:

Individuals will be excluded if any of the following exclusion criteria apply:

* Dementia, fronto temporal dementia (FTD) or other neurodegenerative disorder interfering with compliance.
* Metabolic, neoplastic, or other physically or mentally debilitating disorder.
* 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.
* Use of Vit B3 or blue berry extracts outside the study

Primary outcomes:
measure: Incidence of adverse events
timeFrame: Through study completion, 1 year
Secondary outcomes:
measure: Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
timeFrame: Determined after 12 months of follow-up.
measure: Change in vital capacity
timeFrame: Determined after 12 months of follow-up.
Other outcomes:
measure: Overall survival
timeFrame: Determined after 12 months of follow-up.
Locations:
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Bodø
country: Norway
facility: Nordlandssykehuset HF
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: Kristiansand
country: Norway
facility: Sørlandet sykehus
city: Lillehammer
country: Norway
facility: Sykehuset Innlandet HF
city: Lørenskog
country: Norway
facility: Akershus University Hospital
city: Molde
country: Norway
facility: Helse Møre og Romsdal
city: Namsos
country: Norway
facility: Helse Nord-Trøndelag HF
city: Oslo
country: Norway
facility: Oslo Univerity Hospital
city: Sarpsborg
country: Norway
facility: Sykehuset Østfold HF
city: Skien
country: Norway
facility: Sykehuset i Telemark HF
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
city: Tønsberg
country: Norway
facility: Sykehuset i Vestfold HF
Study officials:
affiliation: Haukeland University Hospital
name: Ole-Bjørn Tysnes
role: PRINCIPAL_INVESTIGATOR
References: