Disease: Parkinson's disease
Type of study: Interventional trial
Coordinating investigators: Geir Olve Skeie & Gard S. Johanson
Study director: Charalampos Tzoulis
Background: Atypical parkinsonian syndromes (APS), including progressive supranuclear palsy (PSP), multiple system atrophy (MSA) and corticobasal syndrome (CBS), are major and entirely unaddressed health challenges. There are currently no treatments able to improve function or delay disease progression in APS, and patients succumb to rapidly increasing disability, with an estimated overall survival of 3-10 years from diagnosis. Despite their devastating health impact, there is currently no clinical treatment research on PSP, MSA, or CBS in Norway, and very few initiatives globally.
Groundbreaking research from the Tzoulis group has nominated the NAD-precursor nicotinamide riboside (NR) as a potential disease-modifying therapy for neurodegenerative parkinsonisms. Motivated by this discovery, we will perform the NADAPT study: a phase-II, double-blind randomised trial of NR in PSP, MSA and CBS. Given the dismal prognosis and complete lack of treatment options for individuals with APS, this trial is both timely and necessary.
The primary objective of the NADAPT study is to determine whether treatment with NR, 3000 mg daily, can delay disease progression in PSP, MSA, and/or CBS.
Design: NADAPT is a phase II double-blind randomised clinical trial of oral NR in APS. Eligible patients will be recruited into three parallel cohorts, including PSP (n=130), MSA (n=165) and CBS (n=30-50). In each cohort, participants will be randomised 1:1 on NR 3000 mg per day or placebo and followed for 78 weeks. Participants will be recruited from Norway, the UK, and France.
Primary endpoint: The between-group (NR vs. placebo) difference in the change from baseline to end of study in disease-specific clinical severity scores (PSPRS, UMSARS, etc.).
Status: The study in ongoing and has 30 participants enrolled.
Participating centres
- Haukeland University Hospital, Bergen
- Akershus University Hospital, Lørenskog
- Oslo University Hospital, Oslo
- Vestre Viken Hospital, Drammen
- University Hospital of North Norway, Tromsø
- Førde Hospital, Førde
- Dr. Karen Herlofson, Arendal
- Haugesund Hospital, Haugesund
- Molde Hospital, Molde
- UCL Queen Square Institute of Neurology, UK
- Pitie Salpetriere Hospital, Paris, France
Funding
- The Norwegian Parkinson’s Disease Association
- KLINBEFORSK
- The Regional Health Authority of Western Norway
- The DAM Foundation
- The Research Council of Norway, Neuro-SysMed
- Haukeland University Hospital
- Participating hospitals