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NOT YET RECRUITING

NCT07692399

PHASE2

Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL

Sponsor: Huashan Hospital

View on ClinicalTrials.gov

Summary

This study is a single-center, prospective, single-arm, self-controlled clinical trial designed to assess the safety and efficacy of edaravone dexborneol sublingual tablets for blood-brain barrier (BBB) dysfunction in patients with CADASIL. The study will enroll approximately 60 participants aged 18 to 80 years with genetically confirmed CADASIL. Participants will be followed for a total duration of 12 months, including two consecutive phases. * Phase 1 is a 6-month natural history observation period, during which participants do not receive the study drug and continue their routine standard care for CADASIL. * Phase 2 is a 6-month drug intervention period, in which participants will receive edaravone dexborneol sublingual tablets to investigate the effect on the BBB water exchange rate (kw), measured by diffusion-prepared pseudo-continuous arterial spin labeling (DP-pCASL) MRI, and to assess potential benefits on stroke risk reduction, cognitive function, and gait performance. The primary endpoint is the change in kw measured by DP-pCASL. Secondary endpoints include the incidence of clinical stroke events; changes in neuropsychological performance, MRI-based CSVD biomarkers, gait and motor assessments, functional disability and activities-of-daily-living scales, and peripheral blood biomarkers. Safety assessments will include adverse events (AEs) and serious adverse events (SAEs).

Official title: Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL: A Single-Center, Prospective, Single-Arm, Self-Controlled Clinical Trial.

Key Details

Gender

All

Age Range

18 Years - 80 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-07

Completion Date

2027-12

Last Updated

2026-07-09

Healthy Volunteers

Conditions

CADASIL

Interventions

DRUG

Edaravone Dexborneol Sublingual Tablets

One tablet (containing edaravone 30 mg and dexborneol 6 mg) is taken sublingually twice daily for the 6-month treatment period.

Inclusion Criteria: * 1\. Genetically Confirmed CADASIL: Diagnosis of CADASIL confirmed by NOTCH3 genetic testing. * 2\. Age: Between 18 and 80 years. * 3\. Lacunar Lesion Requirement: Presence of ≥1 lacune on brain MRI. * 4\. No acute ischemic stroke or intracerebral hemorrhage within the past 3 months. * 5\. Modified Rankin Scale (mRS) score of 0 to 3. * 6\. Contraception Requirements: Women of childbearing potential and male participants with female partners of childbearing potential must agree to use effective contraception during the study and 30 days after the last dose of the investigational drug.Female participants must have a negative pregnancy test before enrollment. * 7\. Informed Consent: Participants or their legal representatives must voluntarily sign an informed consent form (ICF). Exclusion Criteria: * 1\. Presence of other major neurological disorders: Non-vascular white matter diseases (e.g., multiple sclerosis, carbon monoxide encephalopathy), central nervous system infections, Creutzfeldt-Jakob disease, primary Parkinson's disease, traumatic brain injury, or intracranial tumors. * 2\. Severe Liver Dysfunction: Active liver disease (acute hepatitis, chronic active hepatitis, cirrhosis) or ALT/AST \>2× ULN. * 3\. Severe renal impairment (serum creatinine \>1.5× ULN). * 4\. Life Expectancy \<1 year due to severe systemic diseases. * 5\. Contraindications to MRI: Participants with MRI-incompatible implants, severe claustrophobia, or inability to undergo MRI. * 6\. Known Allergies: History of hypersensitivity to Dexborneol, natural borneol, edaravone, or any excipients (e.g., mannitol, copovidone, microcrystalline cellulose, silica, magnesium stearate). * 7\. Pregnancy and Lactation: Pregnant or lactating women, or those planning pregnancy during the study period. * 8\. Participation in Other Clinical Trials * 9\. Other Investigator-Determined Factors: Any other medical, psychological, or social condition that, in the investigator's judgment, makes the patient unsuitable for participation.

Locations (1)

Huashan Hospital, Fudan University

Shanghai, China