The objective of this trial is the global risk-benefit assessment of Cerebrolysin as compared to Placebo in patients with genetically proven CADASIL. In addition, a traditional approach will be taken based on an evaluation of the separate risk and benefit domains in comparison with placebo.
Safety data area collected throughout the study (adverse events, vital signs and laboratory tests) and thereafter in case of ongoing serious adverse events (SAEs) at study endpoint. Optional secondary parameters include analyses of biomarkers (samples of blood, hair, urine, and saliva).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
40 ml Cerebrolysin and 60 ml 0.9% NaCl per day for 4 days every month for 1 year
100 ml 0.9% NaCl per day for 4 days every month for 1 year
Motol University Hospital
Prague, Czechia
Change in cognitive battery (RAVLT)
\- Rey Auditory Verbal Learning Test (AVLT)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (ROCF: Copy, immediate recall)
\- Rey-Osterrieth Complex Figure Test (ROCF): Copy, immediate recall
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (Digit Symbol Coding, subscale of WAIS-PSI)
\- Digit Symbol Coding (subscale of WAIS-PSI)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (Digit Span: Digit backward (subscale of WAIS-WMI)
\- Digit Span: Digit backward (subscale of WAIS-WMI)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (Trail Making Test, Part B)
\- Trail Making Test (Part B)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (ROCF: Delayed recall)
\- Rey-Osterrieth Complex Figure Test (ROCF): Delayed recall
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (Stroop Color and Word Test - Prague Version)
\- Stroop Color and Word Test - Prague Version (word/dots interference)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery (MoCA)
\- Montreal Cognitive Assessment (MoCA)
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Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in mood (Beck Depression Inventory-II)
\- Beck Depression Inventory-II (BDI-II)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in imaging (White matter lesion volume)
\- White matter lesion volume (MRI)
Time frame: Baseline, Month 12, Month 27
Change in cognitive battery, secondary outcome (Spatial Pattern Separation Task)
\- Spatial Pattern Separation Task
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery, secondary outcome (Navigation Test Suite)
\- Navigation Test Suite
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery, secondary outcome (Trail Making Test, Part A)
\- Trail Making Test (Part A)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery, secondary outcome (Stroop Color and Word Test - Prague Version)
\- Stroop Color and Word Test - Prague Version (color-word/dots interference)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery, secondary outcome (Symbol Search, subscale of WAIS-PSI)
\- Symbol Search (subscale of WAIS-PSI)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in cognitive battery, secondary outcome (Digit Span: Digit forward, subscale of WAIS-WMI)
\- Digit Span: Digit forward (subscale of WAIS-WMI)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in mood, secondary outcome (Beck Anxiety Inventory)
\- Beck Anxiety Inventory
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in neurological deficits, secondary outcome (NIH stroke scale)
\- NIH stroke scale (NIHSS)
Time frame: Baseline, Month 6, Month 12, Month 21, Month 27
Change in imaging, secondary outcome (Index of general cortical thinning)
\- Index of general cortical thinning (MRI)
Time frame: Baseline, Month 12, Month 27
Change in imaging, secondary outcome (Post-stroke lacune volume)
\- Post-stroke lacune volume (MRI)
Time frame: Baseline, Month 12, Month 27
Change in biomarker analysis, secondary outcome (Neurofilament light chain)
\- Neurofilament light chain (NFL)
Time frame: Baseline, Month 12, Month 27