CERICA: CERebrolysin In CADASIL
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
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 Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Other: Study group 1 Period 1 (Treatment phase I): Cerebrolysin - Wash out - Period 2 (Treatment phase II): Placebo |
Drug: Cerebrolysin
40 ml Cerebrolysin and 60 ml 0.9% NaCl per day for 4 days every month for 1 year
Other Names:
Drug: 0.9 % NaCl
100 ml 0.9% NaCl per day for 4 days every month for 1 year
Other Names:
|
| Other: Study group 2 Period 1 (Treatment phase I): Placebo - Wash out - Period 2 (Treatment phase II): Cerebrolysin |
Drug: Cerebrolysin
40 ml Cerebrolysin and 60 ml 0.9% NaCl per day for 4 days every month for 1 year
Other Names:
Drug: 0.9 % NaCl
100 ml 0.9% NaCl per day for 4 days every month for 1 year
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Change in cognitive battery [Baseline, Month 6, Month 12, Month 21, Month 27]
Comprise 9 single analysis variables: Auditory Verbal Learning Test (AVLT) Rey-Osterrieth Complex Figure Test (ROCF): Copy, immediate recall Digit Symbol Coding (subscale of WAIS-PSI) Digit Span: Digit backward (subscale of WAIS-WMI) Trail Making Test (Part B); - Rey-Osterrieth Complex Figure Test (ROCF): Delayed recall Stroop Color and Word Test - Victoria Version (word/dots interference) Blue Velvet Arena Test Montreal Cognitive Assessment (MoCA)
- Change in mood [Baseline, Month 6, Month 12, Month 21, Month 27]
- Beck Depression Inventory (BDI)
- Change in imaging [Baseline, Month 12, Month 27]
- White matter lesion volume
Secondary Outcome Measures
- Change in cognitive battery (secondary outcome) [Baseline, Month 6, Month 12, Month 21, Month 27]
Comprise 5 single analysis variables: Trail Making Test (Part A) Stroop Color and Word Test-Victoria Version (color-word/dots interference) Symbol Search (subscale of WAIS-PSI) Digit Span: Digit forward (subscale of WAIS-WMI) NIH stroke scale (NIHSS)
- Change in mood (secondary outcome) [Baseline, Month 6, Month 12, Month 21, Month 27]
- Beck Anxiety Inventory
- Change in imaging (secondary outcome) [Baseline, Month 12, Month 27]
Index of general cortical thinning (MRI) Post-stroke lacune volume (MRI)
Other Outcome Measures
- Experimental: Change in Biomarker analysis [Baseline, Month 12, Month 27]
Biomarker analysis (samples of blood, hair, urine, and saliva)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Patients of ≥18 years of age, all genders
-
Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis
-
MMSE >18
-
Adequate visual, auditory, and language skills (no language interpreter required) to follow study procedures
-
Patient is not of childbearing potential (i.e. women are post-menopausal for two years, surgically sterile, or using adequate method of contraception)
-
Patient participates voluntarily and gave written informed consent
Exclusion Criteria:
-
Any significant neurological disease/conditions other than CADASIL
-
Focal lesions that may be responsible for the cognitive status of the patient (e.g.
infectious disease, space-occupying lesion, normal pressure hydrocephalus)
-
Any other diseases/conditions that may affect compliance with the protocol, such as:
-
severe psychiatric disorders within the last three months
-
delusional symptoms
-
history of schizophrenia, schizoaffective disorder, bipolar affective disorder
-
major depressive disorder newly identified within eight weeks before screening
-
history of alcohol or substance abuse or dependence within the past two years
-
Any circumstances that -in the investigator's opinion- may result in the patient's non-compliance with study procedures, e.g. fragile or thin veins that prevent many i.v. infusions
-
Any other disease/conditions that may affect the safety assessment, such as:
-
history of systemic cancer within the past two years
-
history of myocardial infarction in the past year or unstable or severe cardiovascular disease (including uncontrolled hypertension and/or history of unstable hypertension not compensated by antihypertensive therapy)
-
any clinically significant laboratory abnormalities at screening
-
uncontrolled insulin-requiring diabetes or non-insulin dependent diabetes mellitus (HbA1c >10.0)
-
Use of concomitant medication with neuroprotective/neurotrophic/nootropic effects (e.g. ginkgo biloba, erythropoietin, citicoline, amantadine, piracetam)
-
Any condition that would represent a contraindication for Cerebrolysin administration:
-
hypersensitivity to one of the components of the drug
-
epilepsy
-
severe renal impairment (estimated Glomerular Filtration Rate [eGFR] <30 ml/min/1.73 m2 as assessed at local laboratory within one month before screening)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Motol University Hospital | Praha | Czechia | 150 06 |
Sponsors and Collaborators
- Ever Neuro Pharma GmbH
- idv Datenanalyse & Versuchsplanung
- X Clinical GmbH
Investigators
- Principal Investigator: Aleš Tomek, MUDr., Ph.D., Motol University Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- EVER-CZ-0421
- 2022-002394-29