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CADASIL Research Studies & Clinical Trials

Your participation directly advances our understanding of CADASIL and brings us closer to a cure. Find ongoing studies and learn how to get involved.

CADASIL is a rare disease that has only been identified within the past few decades. As such, there is still significant research to be done to fully understand its pathology, progression, and other distinct aspects.

This research requires willing participants from within the community of CADASIL patients. Below, you can find links to ongoing CADASIL research studies or clinical trials, including information on how your involvement can help us get closer to developing new treatments and eventually a cure.

If your organization or university will be starting a new CADASIL-related research project in the near future, contact us at info@cureCADASIL.org.

We are not aware of CADASIL clinical trials that are currently seeking participants to test treatments (see Active Studies).

Participants can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research. Having an engaged patient community will increase the chances that a pharmaceutical company will want to do clinical trials to test potential therapies.
We are not aware of clinical trials that are currently seeking participants to test treatments for CADASIL. Why aren't there treatment trials? Before we can perform studies to determine whether treatments are safe and effective, a number of important preliminary steps must be taken.

Drug Development Process

Finding Targets and Therapies

Scientific research is needed before drugs and therapies can be tested in humans.

  • Researchers will collect patients' natural history and test results to use with computers/AI, cells, and animal models to identify potential targets for therapies
  • Researchers use human cells to test the potential targets and identify which potential therapies can affect the cellular abnormalities found in CADASIL
Testing Therapeutic Options

Clinical trials involve people who volunteer to test and help find better treatments.

  • Clinical Trial Phases I–III will test drug and therapy safety and efficacy in humans
  • If a therapy is proven safe and effective, it can be approved by the FDA and become available for use in clinical practice
  • After the therapy enters clinical practice, post-market monitoring begins
A Cure for CADASIL
 

Once the disease has been studied, clinical trials have been conducted, and a therapy has been proven safe and effective, we are on our way to treating and curing CADASIL!

Blood samples and other standard assessments from individuals living with CADASIL and from healthy volunteers hold the key to understanding CADASIL.

Enrollment in ongoing studies is crucial!

See the current enrolling studies below.

Studies to Join Now

CZI Study
Patient-Partnered Collaboration
CZI Patient-Partnered Collaboration Study

Lead Investigator: Dr. Fanny Elahi, MD, PhD
Icahn School of Medicine at Mount Sinai

Research Contact:
VascBrain@mssm.edu or CADASIL@mssm.edu

Learn More
CADASIL Consortium Study
Multi-Site Natural History Study
The CADASIL Consortium Study

Lead Investigator: Dr. Jane Paulsen, PhD

Research Contact:
info@CADASIL-Consortium.org
or call: 833-795-3016

Learn More
NIH CADASIL Natural History Study
NIH / NHLBI Study
NIH/NHLBI CADASIL Natural History Study

Lead Investigator:
Elisa Ferrante, PhD
elisa.ferrante@nih.gov

Research Contact:
William Whalen
william.whalen@nih.gov

Learn More

Additional Programs & Resources

cureCADASIL is collaborating with members of the Clinical Genome Resource (ClinGen) team to share genetic and health information from patients affected by CADASIL through the ClinGen Patient Data Sharing Program. This information will be collected through the cureCADASIL Family Registry.

Why is data sharing needed?

When a laboratory finds a genetic change (variant) for the first time, often little is known about it. Even if a genetic variant is well understood, data sharing helps increase understanding of a condition's features, which may help identify possible interventions and treatments. The more information collected, the better researchers will understand how genes affect health and, ultimately, how best to care for patients.

Complete these 3 steps to participate:

1. Login to your account (or create an account if you have not) at: cureCADASIL Family Registry.

2. Review and complete the ClinGen Data Sharing Program informed consent.

CADASIL Community Natural History Study — Learn more about this important cureCADASIL-supported study tracking disease progression to inform future clinical trials.

cureCADASIL is aware of three CADASIL Tissue Banks in the US supported by brain and tissue donations by surviving families of CADASIL patients. For researchers interested in CADASIL tissue for research, please contact cureCADASIL. For patients or families interested in donating a loved one's brain or tissue for CADASIL research please contact one of the following:

Michael M. Wang M.D., Ph.D.
University of Michigan, Department of Neurology
VA Ann Arbor Healthcare System, Ann Arbor, MI 48109
Phone: 734-845-5202  |  Email: micwang@umich.edu

Helmi L. Lutsep, M.D.
Oregon Health & Science University, Department of Neurology
VA Portland Health Care System, Portland, OR 97239
Phone: 503-494-7225  |  Email: lutseph@ohsu.edu

We are not aware of clinical trials that are currently seeking participants to test treatments for CADASIL. See Active Studies for the latest information.