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- Svenningsson, P., et al.
(författare)
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A Phase 2a Trial Investigating the Safety and Tolerability of the Novel Cortical Enhancer IRL752 in Parkinson's Disease Dementia
- 2020
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Ingår i: Movement Disorders. - : Wiley. - 0885-3185 .- 1531-8257. ; 35:6, s. 1046-1054
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Tidskriftsartikel (refereegranskat)abstract
- Background IRL752 is a novel small-molecule compound that acts to regioselectively enhance norepinephrine, dopamine, and acetylcholine neurotransmission in the cerebral cortex. Objective The primary objective of the trial was to investigate the safety and tolerability of IRL752 in patients with Parkinson's disease and dementia. Methods Patients with Parkinson's disease and dementia were randomized to IRL752 or placebo treatment (3:1 ratio) for 28 days. The study drug was given as an adjunct treatment to the patients' regular stable antiparkinsonian medication. Dosing was individually titrated for 14 days after which the dose was kept stable for an additional 14 days. Results A total of 32 patients were randomized to treatment, and 29 patients completed the 4-week treatment. Adverse events were generally mild and transient and were mostly reported during the dose titration phase. There were 2 serious adverse events, and none of them were related to the experimental treatment. The average dose achieved in the stable dose phase was 600 mg daily, yielding a 2-hour postdose plasma concentration of about 4 mu M on day 28. Exploratory assessment of secondary outcomes indicated efficacy for symptoms and signs known to be poorly responsive to levodopa. Conclusions IRL752 appears to be safe and well tolerated for a 4-week treatment in patients with Parkinson's disease and dementia. (c) 2020 International Parkinson and Movement Disorder Society
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- von Below, Daniel, et al.
(författare)
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Validation of the Swedish Patient-Reported Outcomes in Parkinson's Disease Scale in Outpatients
- 2023
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Ingår i: Movement Disorders. - 0885-3185. ; 38:9, s. 1668-1678
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Tidskriftsartikel (refereegranskat)abstract
- BackgroundSuccessful treatment of Parkinson's disease (PD) requires symptom monitoring. Patient-Reported Outcomes in Parkinson's Disease (PRO-PD) is a broad scale that covers 35 motor and nonmotor symptoms, but its validation is limited. ObjectiveThe aim was to validate PRO-PD in a random sample of outpatients with PD. MethodsOf 2123 PD patients who visited outpatient clinics in West Sweden over a 12-month period, 25% were randomly selected and invited to participate in a longitudinal observational study. Included patients were assessed at baseline, 1 year, and 3 years, with a subset also assessed at 3 to 6 months. The assessments included PRO-PD, other patient-reported scales, and Clinical Impression of Severity Index for Parkinson's Disease (CISI-PD). ResultsThe study included 286 PD patients. PRO-PD ratings were available from 716 (96%) of 747 study visits. All PRO-PD items exhibited positive skewness without ceiling effects. Internal consistency at baseline was excellent (Cronbach's & alpha;: 0.93). Six-month test-retest reliability was good (intraclass correlation coefficient: 0.87). Convergent validity was good, with correlation coefficients between total PRO-PD and the 8-Item Parkinson's Disease Questionnaire of 0.70, the Non-Motor Symptoms Questionnaire of 0.70, EuroQoL Five-Dimension Five-Level Scale of 0.71, and CISI-PD of 0.69. Median PRO-PD score at baseline was 995 (interquartile range: 613-1399), with a median yearly increase of 71 (interquartile range: -21 to 111). Items representing axial motor symptoms increased most over time. The minimal clinically important change in total score was 119. ConclusionsPRO-PD was found reliable and valid for monitoring symptoms in a representative sample of outpatients with PD.
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