The aim of this phase Ila trial is to provide evidence on safety, tolerability and symptomatic efficacy of the ROCK-inhibitor Fasudil in patients with early Parkinson's disease (PD). Fasudil has shown neuroprotective and pro-regenerative effects, modulated microglial activity and attenuated alpha-synuclein aggregation in PD models in vitro and in vivo. It has been licensed in Japan since 1995 for the treatment of vasospasms and has a beneficial safety profile arguing for its repurposing. Up to 15 trial centers in Germany will recruit patients. Blinded trial medication will be prepared and shipped by the University Pharmacy Leipzig. Fasudil in two dosages or placebo will be administered orally twice daily to 75 early PD patients for a total of 3 weeks. Safety, tolerability and symptomatic efficacy endpoints will be assessed up to 4 weeks after end of treatment. Its well-known safety profile and the lack of disease-modifying treatments for PD justifies its use in patients with early Parkinson's disease. ROCK-PD is a prerequisite for subsequent long-term clinical trials assessing disease-modification in PD in addition to symptomatic efficacy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
75
Duration of intervention per patient: 22 days; Application scheme: one dose on day 1, two doses on days 2 - 21, one dose on day 22.
0.05 ml Quinine dihydrochloride solution (from Quinina Labesfal) in screw flask supplemented with 30 ml Glucose 40% solution from miniplasco directly before use
Technische Universität München, Klinikum rechts der Isar, Klinik und Poliklinik für Neurologie
Munich, Germany
RECRUITINGCombined occurrence of intolerance and/or occurrence of self-reported and pre-defined treatment-related SAEs
Combined occurrence of intolerance (termination of treatment because of treatment-related AE) and/or occurrence of self-reported and pre-defined (laboratory, vital signs) treatment-related SAEs
Time frame: from day 1 to day 22
Occurrence of intolerance
Occurrence of intolerance (termination of treatment because of treatment-related AE)
Time frame: from day 1 to day 22
Occurrence of self-reported and pre-defined treatment-related SAEs
Occurrence of self-reported and pre-defined (laboratory, vital signs) treatment-related SAEs
Time frame: from day 1 to day 22, and day 1 to day 50
Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
the change of MDS-Unified PD Rating Scale (MDS-UPDRS) each part I to IV, min-max: 0-260, 0 indicating no disability and 260 indicating total disability, Part I: Nonmotor experiences of daily living: 13 items. Score range: 0-52; Part II: Motor experiences of daily living: 13 items. Score range: 0-52; Part III: Motor examination: 18 items. Score range: 0-132; Part IV: Motor complications: 6 items. Score range: 0-24; Each item has 0-4 ratings: 0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe).
Time frame: part I and II from day 1 to day 22, and day 1 to day 50; time frame: part III and IV from day 1 to day 10, day 1 to day 22, and day 1 to day 50
MDS-Unified PD Rating Scale (MDS-UPDRS) score
the change of MDS-Unified PD Rating Scale (MDS-UPDRS) score part I to IV, min-max: 0-260, 0 indicating no disability and 260 indicating total disability, Part I: Nonmotor experiences of daily living: 13 items. Score range: 0-52; Part II: Motor experiences of daily living: 13 items. Score range: 0-52; Part III: Motor examination: 18 items. Score range: 0-132; Part IV: Motor complications: 6 items. Score range: 0-24; Each item has 0-4 ratings: 0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe).
Time frame: from day 1 to day 22, and day 1 to day 50
Parkinson's Disease Questionnaire (PDQ-8)
the change of 8-item PD Quality of Life Scale (PDQ-8), min-max: 0-32, 0 no and 32 max
Time frame: from day 1 to day 22, and day 1 to day 50
Non-Motor Symptom Questionnaire (NMSQuest)
the change of PD Non-Motor Symptom Questionnaire (NMSQuest), min-max: 0-30, 0 no and 30 max
Time frame: from day 1 to day 10, day 1 to day 22, and day 1 to day 50
Montreal Cognitive Assessment (MoCA)
the change of Montreal Cognitive Assessment (MoCA), Montreal Cognitive Assessment MoCA: min-max: 0 (worse) -30 (better outcome)
Time frame: from day 1 to day 22, and day 1 to day 50
Beck's Depression Inventory II (BDI-II)
the change of Becks depression inventory-II (BDI-II), min-max: 0-63, 0 no and 63 max
Time frame: from day 1 to day 22, and day 1 to day 50
Clinician Global Impression of Improvement (CGI-I) and Patient Global Impression of Improvement (PGI-I)
CGI-I \[clinician\]: min-max: 1-7, min-max: 1 (better) -7 (worse outcome) PGI-I \[patient\]: min-max: 1-7, min-max: 1 (better) -7 (worse outcome)
Time frame: at day 10, 22 and day 50
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