Disease: ALS
Type of study: Observational trial
Coordinating investigators: Tale L. Bjerknes & Ole-Bjørn Tysnes
Study directors: Ole-Bjørn Tysnes & Charalampos Tzoulis
Background: Patients with ALS have highly variable clinical phenotypes. Symptoms most commonly start in the extremities, although ~25% of cases exhibit predominantly bulbar symptoms at disease onset, including dysphagia and dysarthria. Extraocular and sphincter muscles are generally spared until late in the disease course. Survival is highly variable, as respiratory failure and death occurs on average 2-3 years after diagnosis, but 5-10% of patients survive 10 years post-diagnosis. Overall, it has not been possible to distinguish familial and sporadic ALS based on clinical phenotypes.
To address the probable heterogeneity of mitochondrial function in ALS, Neuro-SysMed aims to conduct STRAT-ALS, a longitudinal cohort study to stratify ALS according to underlying biological mechanisms, so that tailored treatments can be developed and applied. Initially, the focus will be mitochondrial markers, based on well-established techniques and preliminary results from studies on Parkinson’s disease. The study will probably start in 2024.
Primary objective: To stratify ALS according to underlying molecular disease mechanisms and develop clinically applicable biomarkers enabling: (i) the classification of patients for participation in targeted clinical trials and (ii) monitoring of treatment efficacy in targeted clinical trials.
Design: STRAT-ALS is a prospective, longitudinal observational cohort study. Participants will be followed longitudinally with systematic clinical assessment, neuroimaging, collection of biological material, and postmortem brain collection.
Status: STRAT-ALS protocols were finalised in 2022 and the study is expected to start in 2024. Only patients from Haukeland University Hospital will participate.
Participating centre
- Haukeland University Hospital, Bergen
Funding
- The Regional Health Authority of Western Norway (Helse Vest), postdoc for Tale L. Bjerknes
- The Research Council of Norway, Neuro-SysMed
- Haukeland University Hospital
- The University of Bergen
- Donations from Nils Arne Morka