NCT07692399Not yet recruitingPhase 2
Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL
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.
Source: ClinicalTrials.gov · Last updated 9 Jul 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 60 (estimated)
- First submitted
- 29 Jun 2026
- Start date
- Jul 2026
- Primary completion date
- Dec 2027
- Lead sponsor
- Huashan Hospital
- NCT number
- NCT07692399
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 60
- From Jul 2026 to the primary completion date, Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 5\. Contraindications to MRI: Participants with MRI-incompatible implants, severe claustrophobia, or inability to undergo MRI.
Other illness
- 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.
- 4\. Life Expectancy \<1 year due to severe systemic diseases.
- 9\. Other Investigator-Determined Factors: Any other medical, psychological, or social condition that, in the investigator's judgment, makes the patient unsuitable for participation.
Lab values
- 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).
- 6\. Known Allergies: History of hypersensitivity to Dexborneol, natural borneol, edaravone, or any excipients (e.g., mannitol, copovidone, microcrystalline cellulose, silica, magnesium stearate).
Other
- 7\. Pregnancy and Lactation: Pregnant or lactating women, or those planning pregnancy during the study period.
- 8\. Participation in Other Clinical Trials
Who the study is looking for
- 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).
Age in the registry: 18 years to 80 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Huashan Hospital, Fudan University Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.