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

Overview

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.

Interventions

• Dietary supplement EH301 (Nicotinamide Riboside/Pterostilbene)
  For this study EH301 is defined as Investigational Product(s) (IP). The ALS extension study is an open label study with active treatment of all patients thet have passed through the NO-ALS trial.

Primary outcome measures

• Incidence of adverse events [Time frame: Through study completion, 1 year]

Secondary outcome measures (2)

• Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) [Time frame: Determined after 12 months of follow-up.]
• Change in vital capacity [Time frame: Determined after 12 months of follow-up.]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

Norway · 17 centers

• Haukeland University Hospital — Bergen
• Nordlandssykehuset HF — Bodø
• Vestre Viken HF — Drammen
• Helse Førde HF — Førde
• Helse Fonna HF — Haugesund
• Sørlandet sykehus — Kristiansand
• Sykehuset Innlandet HF — Lillehammer
• Akershus University Hospital — Lørenskog
• … and 9 more centers

Identifiers

NCT: NCT05095571 · 266376

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗