RecruitingNot Applicable

Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL) Study

United States660 participantsStarted 2022-06-03

Plain-language summary

This is an observational study to better understand the risk factors and progression of CADASIL, a leading cause of vascular cognitive impairment and dementia (VCID). 575 participants will be enrolled and can expect to be on study for up to 5 years.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Must be at least 18 years old
. Positive NOTCH3 genetic testing; OR a positive skin biopsy; OR a willingness to have a NOTCH3 genetic test completed prior to enrolling AND are at-risk for, or diagnosed clinically with, CADASIL
. Willing to commit to three in-person visits (a baseline visit, an 18-month follow-up, and a 36-month follow-up) and to remote visits as needed by phone, email, mail or internet
. Willing to provide documentation of all current medications to study team
. Willing and able to undergo an MRI scan and blood draw at each in-person visit
. Must have a designated "study companion"
. A functional capacity less than 4 on the Modified Rankin Scale

Exclusion criteria

. History of severe learning disability, intellectual disability, or other neurological disease or event not attributable to CADASIL
. History of serious alcohol or drug abuse within the past year
. Unwilling to undergo NOTCH3 genetic testing if there is no test on file

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Performance Measured by Change in Cognitive Executive Function Composite Z-Score

Timeframe: baseline and 36 months

Change in total brain volume between baseline and 36 months

Timeframe: baseline and 36 months

Change in total cerebral spinal volume between baseline and 36 months

Timeframe: baseline and 36 months

Percent change in brain connectivity from baseline to 36 months

Timeframe: baseline and 36 months

Change in Neurofilament Light (Nfl)

Timeframe: baseline and 36 months

Change in World Health Organization Disability Assessment Schedule (WHODAS) Score

Timeframe: baseline and 36 months

Change in CADASIL Severity Score

Timeframe: baseline and 36 months

Trial details

NCT IDNCT05677880

SponsorUniversity of Wisconsin, Madison

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-06

Contact for this trial

Cadasil Consortium

1-833-795-3016 info@cadasil-consortium.org

View on ClinicalTrials.gov More CADASIL trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Must be at least 18 years old
. Positive NOTCH3 genetic testing; OR a positive skin biopsy; OR a willingness to have a NOTCH3 genetic test completed prior to enrolling AND are at-risk for, or diagnosed clinically with, CADASIL
. Willing to commit to three in-person visits (a baseline visit, an 18-month follow-up, and a 36-month follow-up) and to remote visits as needed by phone, email, mail or internet
. Willing to provide documentation of all current medications to study team
. Willing and able to undergo an MRI scan and blood draw at each in-person visit
. Must have a designated "study companion"
. A functional capacity less than 4 on the Modified Rankin Scale

Exclusion criteria

. History of severe learning disability, intellectual disability, or other neurological disease or event not attributable to CADASIL
. History of serious alcohol or drug abuse within the past year
. Unwilling to undergo NOTCH3 genetic testing if there is no test on file

What they're measuring

Performance Measured by Change in Cognitive Executive Function Composite Z-Score

Timeframe: baseline and 36 months

Change in total brain volume between baseline and 36 months

Timeframe: baseline and 36 months

Change in total cerebral spinal volume between baseline and 36 months

Timeframe: baseline and 36 months

Percent change in brain connectivity from baseline to 36 months

Timeframe: baseline and 36 months

Change in Neurofilament Light (Nfl)

Timeframe: baseline and 36 months

Change in World Health Organization Disability Assessment Schedule (WHODAS) Score

Timeframe: baseline and 36 months

Change in CADASIL Severity Score

Timeframe: baseline and 36 months