CERebrolysin In CADASIL

What Was Tested

The trial (phase 2) is built around Cerebrolysin.

It had two arms (groups): Study group 1 (other) and Study group 2 (other).

Cerebrolysin - Placebo.

Placebo - Cerebrolysin.

The registry classes the interventions as drug.

More on the Plan

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).

Who Took Part

The registry lists 30 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Patients of ≥18 years of age, all genders. Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis. 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.

The full record lists 6 inclusion and 19 exclusion criteria; among the exclusions: focal lesions that may be responsible for the cognitive status of the patient (e.g; any other diseases/conditions that may affect compliance with the protocol, such as:; severe psychiatric disorders within the last three months.

How the Study Was Run

The set-up was randomized, crossover and quadruple-blind.

The participants were allocated by chance, so the Cerebrolysin group and the comparison group should start out alike.

Each person switched between Cerebrolysin and the comparison at some point, usually with a gap in between.

Neither the participants nor the staff measuring them knew who was on Cerebrolysin, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2023-11-29 with completion listed for 2026-03-31.

It ran at 1 site in Czechia.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change in cognitive battery (RAVLT) (time frame: Baseline, Month 6, Month 12, Month 21, Month 27). Change in cognitive battery (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) (time frame: Baseline, Month 6, Month 12, Month 21, Month 27).

It also tracked 11 secondary outcomes, for example Change in cognitive battery, secondary outcome (Spatial Pattern Separation Task), Change in cognitive battery, secondary outcome (Navigation Test Suite), Change in cognitive battery, secondary outcome (Trail Making Test, Part A), Change in cognitive battery, secondary outcome (Stroop Color and Word Test - Prague Version), Change in cognitive battery, secondary outcome (Symbol Search, subscale of WAIS-PSI) and Change in cognitive battery, secondary outcome (Digit Span: Digit forward, subscale of WAIS-WMI).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Cerebrolysin in 30 people.

Its size is small, so on its own it can only give an early look.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Cerebrolysin compound page, all human trials.