CADASILPhase 2NCT07692399
Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL
A Phase 2 study testing Edaravone Dexborneol Sublingual Tablets in people with CADASIL, run by Huashan Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- CADASIL
- What is being tested?
- Edaravone Dexborneol Sublingual Tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- Jul 2026to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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).
Show all 9Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
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.
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.
Read more
- 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).
Read the detailed description
This study is a single-center, prospective, single-arm, self-controlled clinical trial evaluating the safety and efficacy of edaravone dexborneol sublingual tablets for blood-brain barrier (BBB) dysfunction in CADASIL patients.
- Background and Rationale CADASIL is the most common monogenic form of cerebral small vessel disease (CSVD), caused by pathogenic mutations in the NOTCH3 gene. BBB dysfunction is increasingly recognized as one of the core pathophysiological mechanisms in CADASIL, contributing to white matter injury, progressive cognitive decline, and recurrent stroke, and represents a potential therapeutic target. Edaravone Dexborneol, a novel free radical scavenger and anti-inflammatory agent, has shown neuroprotective effects in preclinical models and clinical trials for ischemic stroke. The TASTE-SL trial demonstrated that edaravone dexborneol improved 90-day functional outcomes in patients with acute ischemic stroke. Based on these findings, this study is designed to systematically assess the efficacy and safety of edaravone dexborneol sublingual tablets in ameliorating BBB dysfunction in patients with CADASIL, and to investigate their potential benefits in reducing stroke risk and improving cognitive function.
- Study Design and Methods A total of 60 participants aged 18 to 80 years with genetically confirmed CADASIL will be recruited. The primary endpoint is the change in the BBB water-exchange rate (kw), measured by diffusion-prepared pseudo-continuous arterial spin labeling (DP-pCASL) MRI. The trial consists of two sequential phases: during the first 6 months (natural disease observation period), participants do not receive the study drug and continue their routine standard care for CADASIL, allowing for the characterization of intra-individual natural variability of kw. During the subsequent 6 months (treatment period), all participants will receive edaravone dexborneol sublingual tablets in addition to their routine standard care, and treatment-related changes in kw will be evaluated. All participants will undergo multimodal MRI scanning at baseline, month 6, and month 12, along with comprehensive assessments including cognitive testing, clinical scales, and peripheral blood biomarkers.
- Significance This study is expected to fill a significant gap in pharmacological intervention research for CADASIL and provide clinical evidence supporting the potential extension of edaravone dexborneol to genetically mediated small vessel disease.
- Conditions
- CADASIL
- Being tested
- Edaravone Dexborneol Sublingual Tablets
- Medical subject headings
- CADASIL
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Edaravone Dexborneol Sublingual TabletsDrug
One tablet (containing edaravone 30 mg and dexborneol 6 mg) is taken sublingually twice daily for the 6-month treatment period.
| Study group | Role | What they receive |
|---|---|---|
| Edaravone Dexborneol Sublingual Tablets | Experimental | All enrolled participants follow a two-phase, self-controlled schedule. During the first 6 months (natural history observation period), participants do not receive the study drug and continue their routine standard care for CADASIL. During the subsequent 6 months (treatment period), participants receive edaravone dexborneol sublingual tablet in addition to their routine standard care. Receives: Drug: Edaravone Dexborneol Sublingual Tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in whole-brain blood-brain barrier water exchange rate (kw) measured by DP-pCASL MRI
The blood-brain barrier (BBB) water exchange rate (kw), an imaging marker of BBB function, is quantified on whole-brain diffusion-prepared pseudo-continuous arterial spin labeling (DP-pCASL) MRI at baseline, month 6, and month 12. All participants are untreated during the first 6 months (observation period) and receive edaravone dexborneol sublingual tablets during the subsequent 6 months (treatment period). The change in kw during the observation period (baseline to Month 6) is compared with the change during the treatment period (Month 6 to Month 12) to distinguish the natural trajectory of BBB dysfunction from a potential treatment effect.
Measured: Baseline, Month 6, and Month 12
Secondary outcomes (34)
Clinical Stroke Events
Number and proportion of participants experiencing any stroke event, including ischemic stroke and hemorrhagic stroke, recorded throughout the study.
Measured: 12 months
Change in Montreal Cognitive Assessment (MoCA) Score
The Montreal Cognitive Assessment (MoCA) is a cognitive screening tool that assesses multiple domains, including attention, concentration, executive function, memory, language, visuospatial skills, abstract thinking, calculation, and orientation. Scores range from 0 to 30, with higher scores indicating better cognitive function.
Measured: Baseline, Month 6, and Month 12
Change in Auditory Verbal Learning Test (AVLT) Score
Verbal episodic memory is assessed using the Huashan version of the Auditory Verbal Learning Test (AVLT-H). The test uses a list of 12 words from three semantic categories (4 words per category). The examiner reads the list aloud and the participant recalls the words immediately; this is repeated for three consecutive trials. The immediate recall score is the sum of correctly recalled words across the three trials, ranging from 0 to 36, with higher scores indicating better memory function.
Measured: Baseline, Month 6, and Month 12
Change in Boston Naming Test Score
The Boston Naming Test (BNT) is a widely used neuropsychological assessment that measures confrontation naming-the ability to retrieve and produce the correct name for a visually presented object. Boston Naming Test includes 30 items and evaluates language through measuring the total of correct responses. The minimum score is 0, and the maximum score is 30. Higher score indicates better language.
Measured: Baseline, Month 6, and Month 12
Change in Stroop Color-Word Test completion time
Inhibitory function and executive control are assessed using the Stroop Color-Word Test. In the color-word interference condition, participants name the ink color of color words while inhibiting the automatic tendency to read the word. The outcome is the completion time in seconds; shorter times indicate better executive function.
Measured: Baseline, Month 6, and Month 12
Change in Stroop Color-Word Test number of correct responses
Inhibitory function and executive control are assessed using the Stroop Color-Word Test. In the color-word interference condition, participants name the ink color of color words while inhibiting the automatic tendency to read the word. The outcome is the number of correct responses, ranging from 0 to 50, with higher scores indicating better executive function.
Measured: Baseline, Month 6, and Month 12
Change in Symbol Digit Modalities Test (SDMT) Score
The total number of correct symbol-digit pairings completed within 90 seconds (common score range: 0-105), where a higher score indicates better processing speed, sustained attention, and visual scanning.
Measured: Baseline, Month 6, and Month 12
Change in Trail Making Test (TMT) Score
The Trail Making Test (TMT) is a widely used neuropsychological test that measures processing speed, visual attention, sequencing, mental flexibility, and executive function. The score is the time it takes to complete the task. Lower times are better, while longer times indicate greater impairment.
Measured: Baseline, Month 6, and Month 12
Change in Digit Span Test (DST) Score
Digit Span Test (DST) consists of forward and backward subtests, that respectively assess attention and executive function through measuring the number of correct digit sequences. The minimum score is 0 , and the maximum scores are respectively 10 and 9 for forward and backward subtests. The total score is the sum of the forward and backward subtests, ranging from 0 to 19, with higher scores indicating better attention and working memory.
Measured: Baseline, Month 6, and Month 12
Change in Hamilton Anxiety Rating Scale (HAMA) score
The Hamilton Anxiety Rating Scale (HAMA) is a clinician-administered scale used to assess the severity of anxiety symptoms. The total score ranges from 0 to 56, with higher scores indicating more severe anxiety symptoms.
Measured: Baseline, Month 6, and Month 12
Change in Hamilton Depression Rating Scale (HAMD) score
The Hamilton Depression Rating Scale (HAMD) is a widely used clinical tool for assessing the severity of depression. It consists of multiple items that evaluate various aspects of depressive symptoms, including mood, guilt, suicidal ideation, work and interest, among others. It is scored on a scale from 0 to 52, with higher scores indicating more severe symptoms.
Measured: Baseline, Month 6, and Month 12
Change in number of lacunes
The change in the number of lacunes on brain MRI, assessed using T1-weighted, T2-weighted, and FLAIR sequences. Lacunes are markers of cerebral small vessel disease; an increased number may indicate progression of vascular pathology.
Measured: Baseline, Month 6, and Month 12
Change in Number of Microbleeds
The change in the number of cerebral microbleeds on brain MRI, assessed using susceptibility-weighted imaging (SWI) or T2*-weighted gradient-echo imaging. Cerebral microbleeds are markers of cerebral small vessel disease; an increased burden may indicate worsening vascular pathology.
Measured: Baseline, Month 6, and Month 12
Change in White Matter Hyperintensity Volume
The change in white matter hyperintensity (WMH) volume, assessed using quantitative MRI analysis and reported in milliliters (mL). This measure evaluates the impact of treatment on the progression of cerebral small vessel disease. A smaller increase or a decrease in WMH volume suggests a treatment benefit.
Measured: Baseline, Month 6, and Month 12
Change in perivascular space (PVS) score
The change in the burden of enlarged perivascular spaces on brain MRI, rated on a validated visual rating scale (0 to 4) in the basal ganglia and centrum semiovale. Enlarged perivascular spaces are markers of cerebral small vessel disease; a higher score may indicate worsening vascular pathology.
Measured: Baseline, Month 6, and Month 12
Change in total brain volume
The change in total brain volume on brain MRI, assessed using quantitative volumetric analysis of 3D T1-weighted images and reported in milliliters (mL). Loss of brain volume reflects atrophy associated with cerebral small vessel disease; a greater reduction in brain volume may indicate disease progression.
Measured: Baseline, Month 6, and Month 12
Change in fractional anisotropy (FA)
The change in fractional anisotropy (FA) derived from diffusion tensor imaging (DTI), an index of white matter microstructural integrity. A decrease in FA reflects loss of white matter integrity and may indicate progression of cerebral small vessel disease.
Measured: Baseline, Month 6, and Month 12
Change in mean diffusivity (MD)
The change in mean diffusivity (MD) derived from diffusion tensor imaging (DTI). An increase in MD reflects loss of white matter microstructural integrity and may indicate progression of cerebral small vessel disease.
Measured: Baseline, Month 6, and Month 12
Change in peak width of skeletonized mean diffusivity (PSMD)
The change in peak width of skeletonized mean diffusivity (PSMD) derived from diffusion tensor imaging (DTI), a fully automated marker of cerebral small vessel disease. An increase in PSMD may indicate worsening white matter damage and disease progression.
Measured: Baseline, Month 6, and Month 12
Change in Tinetti Balance and Gait Assessment score
Assessed using the Tinetti Balance and Gait Evaluation. The scale consists of a balance section (9 items, max 16 points) and a gait section (7 items, max 12 points). Total scores range from 0 to 28, with higher scores indicating better balance and gait, and lower fall risk.
Measured: Baseline, Month 6, and Month 12
Changes in Short Physical Performance Battery (SPPB) score
Changes in Short Physical Performance Battery (SPPB) score (range 0-12, with higher score indicating better physical performance).
Measured: Baseline, Month 6, and Month 12
Change in modified Rankin Scale (mRS) score
The change in modified Rankin Scale (mRS) score. The mRS is a widely used functional outcome measure that assesses the degree of disability or dependence in daily activities. The scale ranges from 0 (no symptoms) to 6 (death), with higher scores indicating greater disability. An increase in mRS score suggests a worsening of functional status.
Measured: Baseline, Month 6, and Month 12
Change in Instrumental Activities of Daily Living (IADL) scale score
The change in Lawton-Brody Instrumental Activities of Daily Living (IADL) scale score. IADLs are activities that support daily life and involve interacting with one's environment, such as managing finances, medication and health, shopping, meal preparation, housekeeping, laundry, communication, and community mobility. The scale is scored from 0 to 8, with higher scores indicating greater functional independence.
Measured: Baseline, Month 6, and Month 12
Change in serum neurofilament light chain (NfL)
The change in serum neurofilament light chain (NfL) concentration. NfL is a biomarker of neuroaxonal injury; higher levels may indicate greater neuronal damage.
Measured: Baseline, Month 6, and Month 12
Change in serum high-sensitivity C-reactive protein (hs-CRP)
The change in serum high-sensitivity C-reactive protein (hs-CRP) concentration. hs-CRP is a marker of systemic inflammation; higher levels may indicate a greater inflammatory burden.
Measured: Baseline, Month 6, and Month 12
Change in serum interleukin-6 (IL-6)
The change in serum interleukin-6 (IL-6) concentration. IL-6 is a pro-inflammatory cytokine; higher levels may indicate a greater inflammatory burden.
Measured: Baseline, Month 6, and Month 12
Change in serum interleukin-8 (IL-8)
The change in serum interleukin-8 (IL-8) concentration. IL-8 is a pro-inflammatory chemokine; higher levels may indicate a greater inflammatory burden.
Measured: Baseline, Month 6, and Month 12
Change in serum interleukin-10 (IL-10)
The change in serum interleukin-10 (IL-10) concentration. IL-10 is an anti-inflammatory cytokine; higher levels reflect an anti-inflammatory response.
Measured: Baseline, Month 6, and Month 12
Change in serum tumor necrosis factor-alpha (TNF-α)
The change in serum tumor necrosis factor-alpha (TNF-α) concentration. TNF-α is a pro-inflammatory cytokine; higher levels may indicate a greater inflammatory burden.
Measured: Baseline, Month 6, and Month 12
Change in serum glial fibrillary acidic protein (GFAP)
The change in serum glial fibrillary acidic protein (GFAP) concentration. GFAP is a biomarker of astroglial activation and injury; higher levels may indicate greater astrocytic damage.
Measured: Baseline, Month 6, and Month 12
Adverse events
Adverse events, confirmed by the Clinical Event Committee.
Measured: 12 months
Serious adverse events
Serious adverse events, confirmed by the Clinical Event Committee.
Measured: 12 months
Liver function impairment
Number of participants with Alanine Aminotransferase level >2.0×upper limit of the normal
Measured: 12 months
Renal function impairment
Number of participants with serum creatinine >1.5×upper limit of the normal.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2026
- Study startJul 2026 (estimated)
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CADASILStudies of the same condition, open and under way.
- More trials run by Huashan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Huashan Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Jiangsu Simcere Pharmaceutical Co., Ltd.
- Responsible party
- Principal investigator, Xin Cheng, Huashan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07692399
- Sponsor's protocol ID
- KY2026-703
- Status last verified
- Jul 2026
- First submitted
- 29 Jun 2026
- Last updated
- 9 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT07692399
- Acronym / sponsor ID
- KY2026-703
- Conditions
- CADASIL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Huashan HospitalUniversity, hospital or other
- Collaborators
- Jiangsu Simcere Pharmaceutical Co., Ltd.
- Study start
- Jul 2026 (estimated)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 9 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This study is a single-center, prospective, single-arm, self-controlled clinical trial evaluating the safety and efficacy of edaravone dexborneol sublingual tablets for blood-brain barrier (BBB) dysfunction in CADASIL patients.
- Background and Rationale CADASIL is the most common monogenic form of cerebral small vessel disease (CSVD), caused by pathogenic mutations in the NOTCH3 gene. BBB dysfunction is increasingly recognized as one of the core pathophysiological mechanisms in CADASIL, contributing to white matter injury, progressive cognitive decline, and recurrent stroke, and represents a potential therapeutic target. Edaravone Dexborneol, a novel free radical scavenger and anti-inflammatory agent, has shown neuroprotective effects in preclinical models and clinical trials for ischemic stroke. The TASTE-SL trial demonstrated that edaravone dexborneol improved 90-day functional outcomes in patients with acute ischemic stroke. Based on these findings, this study is designed to systematically assess the efficacy and safety of edaravone dexborneol sublingual tablets in ameliorating BBB dysfunction in patients with CADASIL, and to investigate their potential benefits in reducing stroke risk and improving cognitive function.
- Study Design and Methods A total of 60 participants aged 18 to 80 years with genetically confirmed CADASIL will be recruited. The primary endpoint is the change in the BBB water-exchange rate (kw), measured by diffusion-prepared pseudo-continuous arterial spin labeling (DP-pCASL) MRI. The trial consists of two sequential phases: during the first 6 months (natural disease observation period), participants do not receive the study drug and continue their routine standard care for CADASIL, allowing for the characterization of intra-individual natural variability of kw. During the subsequent 6 months (treatment period), all participants will receive edaravone dexborneol sublingual tablets in addition to their routine standard care, and treatment-related changes in kw will be evaluated. All participants will undergo multimodal MRI scanning at baseline, month 6, and month 12, along with comprehensive assessments including cognitive testing, clinical scales, and peripheral blood biomarkers.
- Significance This study is expected to fill a significant gap in pharmacological intervention research for CADASIL and provide clinical evidence supporting the potential extension of edaravone dexborneol to genetically mediated small vessel disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Edaravone Dexborneol Sublingual Tablets | Experimental | Drug: Edaravone Dexborneol Sublingual TabletsAll enrolled participants follow a two-phase, self-controlled schedule. During the first 6 months (natural history observation period), participants do not receive the study drug and continue their routine standard care for CADASIL. During the subsequent 6 months (treatment period), participants receive edaravone dexborneol sublingual tablet in addition to their routine standard care. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Edaravone Dexborneol Sublingual TabletsOne tablet (containing edaravone 30 mg and dexborneol 6 mg) is taken sublingually twice daily for the 6-month treatment period. | Drug | Edaravone Dexborneol Sublingual Tablets |
Primary outcome
Change in whole-brain blood-brain barrier water exchange rate (kw) measured by DP-pCASL MRI
Time frame: Baseline, Month 6, and Month 12
The blood-brain barrier (BBB) water exchange rate (kw), an imaging marker of BBB function, is quantified on whole-brain diffusion-prepared pseudo-continuous arterial spin labeling (DP-pCASL) MRI at baseline, month 6, and month 12. All participants are untreated during the first 6 months (observation period) and receive edaravone dexborneol sublingual tablets during the subsequent 6 months (treatment period). The change in kw during the observation period (baseline to Month 6) is compared with the change during the treatment period (Month 6 to Month 12) to distinguish the natural trajectory of BBB dysfunction from a potential treatment effect.
34 secondary outcomes
Clinical Stroke Events
Time frame: 12 months
Change in Montreal Cognitive Assessment (MoCA) Score
Time frame: Baseline, Month 6, and Month 12
Change in Auditory Verbal Learning Test (AVLT) Score
Time frame: Baseline, Month 6, and Month 12
Change in Boston Naming Test Score
Time frame: Baseline, Month 6, and Month 12
Change in Stroop Color-Word Test completion time
Time frame: Baseline, Month 6, and Month 12
Change in Stroop Color-Word Test number of correct responses
Time frame: Baseline, Month 6, and Month 12
Change in Symbol Digit Modalities Test (SDMT) Score
Time frame: Baseline, Month 6, and Month 12
Change in Trail Making Test (TMT) Score
Time frame: Baseline, Month 6, and Month 12
Change in Digit Span Test (DST) Score
Time frame: Baseline, Month 6, and Month 12
Change in Hamilton Anxiety Rating Scale (HAMA) score
Time frame: Baseline, Month 6, and Month 12
Change in Hamilton Depression Rating Scale (HAMD) score
Time frame: Baseline, Month 6, and Month 12
Change in number of lacunes
Time frame: Baseline, Month 6, and Month 12
Change in Number of Microbleeds
Time frame: Baseline, Month 6, and Month 12
Change in White Matter Hyperintensity Volume
Time frame: Baseline, Month 6, and Month 12
Change in perivascular space (PVS) score
Time frame: Baseline, Month 6, and Month 12
Change in total brain volume
Time frame: Baseline, Month 6, and Month 12
Change in fractional anisotropy (FA)
Time frame: Baseline, Month 6, and Month 12
Change in mean diffusivity (MD)
Time frame: Baseline, Month 6, and Month 12
Change in peak width of skeletonized mean diffusivity (PSMD)
Time frame: Baseline, Month 6, and Month 12
Change in Tinetti Balance and Gait Assessment score
Time frame: Baseline, Month 6, and Month 12
Changes in Short Physical Performance Battery (SPPB) score
Time frame: Baseline, Month 6, and Month 12
Change in modified Rankin Scale (mRS) score
Time frame: Baseline, Month 6, and Month 12
Change in Instrumental Activities of Daily Living (IADL) scale score
Time frame: Baseline, Month 6, and Month 12
Change in serum neurofilament light chain (NfL)
Time frame: Baseline, Month 6, and Month 12
Change in serum high-sensitivity C-reactive protein (hs-CRP)
Time frame: Baseline, Month 6, and Month 12
Change in serum interleukin-6 (IL-6)
Time frame: Baseline, Month 6, and Month 12
Change in serum interleukin-8 (IL-8)
Time frame: Baseline, Month 6, and Month 12
Change in serum interleukin-10 (IL-10)
Time frame: Baseline, Month 6, and Month 12
Change in serum tumor necrosis factor-alpha (TNF-α)
Time frame: Baseline, Month 6, and Month 12
Change in serum glial fibrillary acidic protein (GFAP)
Time frame: Baseline, Month 6, and Month 12
Adverse events
Time frame: 12 months
Serious adverse events
Time frame: 12 months
Liver function impairment
Time frame: 12 months
Renal function impairment
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2026.
- Individual participant dataSharing planned: No
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Florida Cerebrovascular Disease Biorepository and Genomics Center
Cerebrovascular Disease, Ischemic Stroke
RecruitingObservational study
Effect of the Mediterranean Diet in Patients Affected by CADASIL and Cerebral Amyloid Angiopathy.
Cerebral Amyloid Angiopathy, CAA - Cerebral Amyloid Angiopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Edaravone Dexborneol Sublingual Tablets in people with CADASIL, run by Huashan Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 9 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xin Cheng (+86 021-52887145, chengxin@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in whole-brain blood-brain barrier water exchange rate (kw) measured by DP-pCASL MRI (time frame: Baseline, Month 6, and Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2026.