Amyotrophic Lateral Sclerosis
Conditions
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.
Interventions
DIETARY_SUPPLEMENTEH301 (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.
Sponsors
Elysium Health
Haukeland University Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
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.
Eligibility
Sex/Gender
ALL
Age
35 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse events | Through study completion, 1 year | Studies of adverse events including changes in lab parameters induced by high dose oral NR/pterostilbene. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Disease progression as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) | Determined after 12 months of follow-up. | 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 in vital capacity | Determined after 12 months of follow-up. | Vital capacity measured by spirometry. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Overall survival | Determined after 12 months of follow-up. | Defined as how many are alive or not requiring continuous respiratory ventilation life support. |
Countries
Norway
Contacts
Primary ContactOle-Bjørn Tysnes
Outcome results
None listed