CEREBROLYSIN CONCENTRATE for CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy)

Inclusion criteria

• {"criterion_text":"- Patients of ≥18 years of age, all genders"}
• {"criterion_text":"- Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis"}
• {"criterion_text":"- MoCA >11"}
• {"criterion_text":"- Adequate visual, auditory, and language skills (no language interpreter required) to follow study procedures"}
• {"criterion_text":"- Patient is not of childbearing potential (i.e. women are post-menopausal for two years, surgically sterile, or using adequate method of contraception such as hormonal contraception in combination with a barrier method, the use of an intrauterine device or hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence)"}
• {"criterion_text":"- Patient participates voluntarily and gave written informed consent"}

Exclusion criteria

• {"criterion_text":"- Any significant neurological disease/conditions other than CADASIL"}
• {"criterion_text":"- Focal lesions that may be responsible for the cognitive status of the patient (e.g. infectious disease, space-occupying lesion, normal pressure hydrocephalus)"}
• {"criterion_text":"- Any other diseases/conditions that may affect compliance with the protocol, such as: a.\tsevere psychiatric disorders within the last three months b.\tdelusional symptoms c.\thistory of schizophrenia, schizoaffective disorder, bipolar affective disorder d.\tmajor depressive disorder newly identified within eight weeks before screening e.\thistory of alcohol or substance abuse or dependence within the past two years"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Any other disease/conditions that may affect the safety assessment, such as: a.\thistory of systemic cancer within the past two years b.\thistory 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) c.\tany clinically significant laboratory abnormalities at screening d.\tuncontrolled insulin-requiring diabetes or non-insulin dependent diabetes mellitus (HbA1c >87 mmol/mol)"}
• {"criterion_text":"- Use of concomitant medication with neuroprotective/neurotrophic/nootropic effects (e.g. ginkgo biloba, erythropoietin, citicoline, amantadine, piracetam)"}
• {"criterion_text":"- Any condition that would represent a contraindication for Cerebrolysin administration: a.\thypersensitivity to one of the components of the drug b.\tepilepsy c.\tsevere renal impairment (estimated Glomerular Filtration Rate [eGFR] <30 ml/min/1.73 m2 as assessed at local laboratory within one month before screening)"}

Primary endpoints

• {"endpoint_text":"- The primary multidimensional outcome ensemble comprises 10 single analysis variables of three dimensions (cognition, mood, imaging characteristics) assessed during and at the end of treatment phases I and II (at months 6, 12, 21, and 27). Month 12 (end of phase I) and Month 27 (end of phase II) are the primary endpoints of the formal cross-over analysis.","definition_or_measurement_approach":"Ten single analysis variables across three dimensions (cognition, mood, imaging characteristics) assessed during and at the end of treatment phases I and II at months 6, 12, 21 and 27. The formal cross-over analysis uses Month 12 (end of phase I) and Month 27 (end of phase II) as the primary timepoints."}

Czechia

Earliest CTIS Part Ii Submission Date

04-09-2024

Latest Decision Or Authorization Date

19-09-2024

Processing Time Days

15

Number Of Sites

1

Number Of Participants

30

Primary sponsor

Full Name

Ever Neuro Pharma GmbH

Organisation Type

Pharmaceutical company

Country Of Registered Address

Austria