CERebrolysin In CADASIL

Prior treatment

• 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
• Use of concomitant medication with neuroprotective/neurotrophic/nootropic effects (e.g. ginkgo biloba, erythropoietin, citicoline, amantadine, piracetam)

Other illness

• 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)

Lab values

• 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 \>87 mmol/mol)
• 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)

Other

• 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

• Patients of ≥18 years of age, all genders
• Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis
• MoCA \>11
• 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