Randomized, Double‑Blind, Placebo‑Controlled, Multiple‑Dose Study of CV‑01: Safety, Tolerability, PK/PD in Healthy Subjects and Drug‑Resistant Epilepsy

Primary objective: To evaluate the safety and tolerability of multiple ascending oral doses of CV-01 administered twice daily for 14 days in healthy volunteers and in participants with drug resistant epilepsy. Determining tolerability is essential to progress the investigational agent in a population where treatment options are limited.

Secondary objectives include:

• Characterization of the pharmacokinetic profile of CV-01 across dose levels.
• Assessment of the pharmacodynamic response, including changes in seizure‑related biomarkers.
• Exploratory evaluation of seizure frequency and severity in the epilepsy cohort.
• Monitoring of laboratory safety markers and vital‑sign trends.

The sponsor did not provide the total number of participants. The trial population comprised both male and female individuals, including patients diagnosed with drug resistant epilepsy as well as healthy volunteers. Vulnerable persons were eligible for enrollment. Age eligibility was defined by the protocol using categories identified as codes 3 and 4. No additional lifestyle requirements or specific inclusion/exclusion criteria were disclosed.

The study is a Phase 3, randomized, multiple ascending doses trial employing a double‑blind, placebo‑controlled design for a 2‑week treatment period in healthy volunteers, followed by a 2‑week open‑label period in a cohort with drug resistant epilepsy. After an initial screening visit to confirm eligibility, participants undergo a baseline assessment and are assigned to dose cohorts. Oral administrations of the investigational product or matching placebo occur daily for 14 days, with safety, safety, tolerability, pharmacokinetics and pharmacodynamics evaluations performed at scheduled follow‑up visits on Day 7 and Day 14. The open‑label extension for the epilepsy cohort continues for an additional 14 days, after which an end‑of‑study visit collects final safety and efficacy data. Participant involvement spans approximately four weeks, with the overall recruitment period defined from 30 June 2026 to 30 August 2027. Early termination may occur in the event of serious adverse events, significant protocol non‑compliance, pregnancy, or voluntary withdrawal of consent.

No experimental medication, comparator, or standard‑of‑care details were supplied in the source data; consequently, a description of the treatment regimens cannot be provided.

Efficacy will be evaluated using predefined clinical endpoints and assessment schedules as specified in the study protocol for the phase 3 trial in drug‑resistant epilepsy.