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Effects of Pridopidine on Functional Capacity in Early-Stage Participants from the PRIDE-HD Study
- Source :
- Journal of Huntington's Disease
- Publication Year :
- 2020
- Publisher :
- IOS Press, 2020.
-
Abstract
- Background: No pharmacological treatment has been demonstrated to provide a functional benefit for persons with Huntington’s disease (HD). Pridopidine is a sigma-1-receptor agonist shown to have beneficial effects in preclinical models of HD. Objective: To further explore the effect of pridopidine on Total Functional Capacity (TFC) in the recent double-blind, placebo-controlled PRIDE-HD study. Methods: We performed post-hoc analyses to evaluate the effect of pridopidine on TFC at 26 and 52 weeks. Participants were stratified according to baseline TFC score and analyzed using repeated measures (MMRM) and multiple imputation assuming missing not-at-random (MNAR) and worst-case scenarios. Results: The pridopidine 45 mg bid dosage demonstrated a beneficial effect on TFC for the entire population at week 52 of 0.87 (nominal p = 0.0032). The effect was more pronounced for early HD participants (HD1/HD2, TFC = 7–13), with a change from placebo of 1.16 (nominal p = 0.0003). This effect remained nominally significant using multiple imputation with missing not at random assumption as a sensitivity analysis. Responder analyses showed pridopidine 45 mg bid reduced the probability of TFC decline in early HD patients at Week 52 (nominal p = 0.02). Conclusion: Pridopidine 45 mg bid results in a nominally significant reduction in TFC decline at 52 weeks compared to placebo, particularly in patients with early-stage HD.
- Subjects :
- 0301 basic medicine
Research Report
Adult
Male
medicine.medical_specialty
Placebo
Severity of Illness Index
Pharmacological treatment
03 medical and health sciences
Cellular and Molecular Neuroscience
chemistry.chemical_compound
0302 clinical medicine
Huntington's disease
Piperidines
Internal medicine
Activities of Daily Living
medicine
pridopidine
Humans
Receptors, sigma
Stage (cooking)
Beneficial effects
business.industry
Repeated measures design
clinical trial
Middle Aged
medicine.disease
total functional capacity
Pridopidine
Clinical trial
030104 developmental biology
Functional Status
Huntington Disease
chemistry
Female
Neurology (clinical)
business
030217 neurology & neurosurgery
Huntington’s disease
Subjects
Details
- Language :
- English
- ISSN :
- 18796400 and 18796397
- Volume :
- 9
- Issue :
- 4
- Database :
- OpenAIRE
- Journal :
- Journal of Huntington's Disease
- Accession number :
- edsair.doi.dedup.....7f3c11384e608a711f1801f09f6f7bd9