Skip to content

Walk N’ Roll season is upon us! Find an event near you.  LEARN MORE!

Natural history study and biomarker identification

To accelerate treatment development

Our Mission

To better understand the factors that determine disease progression among Cerebellar Ataxia with the goal of improving the understanding of the disease process, current treatments, and development of disease-modifying therapies

Patients and Families

Learn more about how you can get involved in the CRC-SCA Natural History Study.

Academic Research Community

General information for researchers working with Ataxia.

Interested Industry

Introductory information for industry partners that are exploring Ataxia therapies.

CRC-SCA Site Log In

For PI’s and Coordinators of CRC-SCA Sites.

CRC-SCA Natural History Study

A natural history study collects data that shows how a specific disease progresses in individuals over time. Natural history studies are essential for developing clinical trial designs that will facilitate drug development. You may also hear the terms ‘observational study’ or ‘observational clinical trial’ used to describe a natural history study.

The natural history of the SCAs and factors that may modify them are not completely understood. There is a need to define natural history in geographically distinct areas and develop additional methods to document progression that may be more sensitive and reliable. There is also limited knowledge of factors that may modify symptoms of SCA. 

The CRC-SCA natural history study and biomarker development is growing and adapting during this exciting new era in ataxia research as pharmaceuticals are advancing to therapy development for the ataxias. CRC-SCA plans to add specific clinical end-point assessments and biospecimen collection. This will increase the strength of this study allowing the consortium to partner with stakeholders in a meaningful way that will include patients as research collaborators.

The Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) continues to recruit research participants who have a confirmed diagnosis of SCA 1, 2, 3, 6, 7, 8, 10, 27B, or RFC1/CANVAS. This is an opportunity for anyone in the United States and Canada with those forms of SCA at any stage of the disease to participate.

You can use our new CRC-SCA Eligibility Screening Tool to confirm you are eligible to participate in this natural history study.

Contact the research coordinator at a site near you to register to participate in the CRC-SCA.

Study Leadership

Study Co-Chair

Liana Rosenthal, MD, PhD
Johns Hopkins University,
Baltimore, MD

Study Co-Chair

Vikram Shakkottai, MD, PhD
University of Texas,   
Southwestern Medical Center       
Dallas, TX

Study Co-Chair

Sheng-Han Kuo, MD
Columbia University,
New York City, NY

Protocol Lead

External Partner Collaboration Lead

Frank Ferrari
frankfer@med.umich.edu

Process Improvement and Patient Engagement Lead

Nadia Amokrane
Na2855@cumc.columbia.edu

US Site Map

US Sites & Coordinators

Columbia University

Drs. Sheng Han Kuo and Chih-Chun Lin

Lead Coordinator:
Nadia Amokrane
Na2855@cumc.columbia.edu

Emory University

Dr. George “Chip” Wilmot

Lead Coordinator:
Emilee Wehunt
emorga9@emory.edu

Houston Methodist Research Institute

Drs. Tetsuo Ashizawa and Andrew Billnitzer

Lead Coordinator:
Nijah Russell
nrussell@houstonmethodist.org

Johns Hopkins University

Drs. Liana Rosenthal and Chiadikaobi Onyike

Lead Coordinator:
Vanessa Johnson
vjohns23@jhmi.edu

Massachusetts General

Drs. Jeremy Schmahmann and Anoopum Gupta

Lead Coordinator:
Jason MacMore
jmacmore@mgh.harvard.edu

Northwestern University

Drs. Puneet Opal and Rizwan Akhtar

Lead Coordinator:
Justin Wong
justin.wong@northwestern.edu

University of California - Los Angeles

Dr. Susan Perlman

Lead Coordinator:
Aaron Fisher
ADFisher@mednet.ucla.edu

University of California - San Francisco

Drs. Cameron Dietiker and Michael Geschwind

Lead Coordinator:
Chelsea Chen
Chelsea.Chen@ucsf.edu

University of Chicago

Dr. Christopher Gomez

Lead Coordinator:
Hannah Casey
hannahcasey@uchicago.edu

University of Florida

Drs. SH Subramony and Matthew Burns

Lead Coordinator:
Jennifer Argudo
jennifer.argudo@neurology.ufl.edu 

University of Michigan

Drs. Henry Paulson and Sharan Srinivasan

Lead Coordinator:
Frank Ferrari
frankfer@med.umich.edu

University of Pennsylvania

Dr. Ali Hamedani

Lead Coordinator:
Anne Beckett-Fedarko
anne.beckett-fedarko@pennmedicinie.upenn.edu 

University of South Florida

Dr. Theresa Zesiewicz

Lead Coordinator:
Julia Tenorio-Ibanez
juliatenorio@usf.edu

University of Texas, Southwestern Medical Center

Dr. Vikram Shakkottai

Lead Coordinator:
Jan Cameron Watts
jan.cameronwatts@utsouthwestern.edu

University of Washington

Dr. Marie Davis

Lead Coordinator:
Sarah Simon
ssimon3@uw.edu

Canada Site & Coordinators

University of Montreal

Montréal, Québec, Canada

Dr. Antoine Duquette

Lead Coordinator:
Martine Comeau
martine.comeau.chum@ssss.gouv.qc.ca

Funding

The CRC-SCA is supported by funding from: the Gordon and Marilyn Macklin Foundation, NAF, NAF’s Drug Development Collaborative, Columbia University, and Ataxia Canada. NAF extends its appreciation to these partners for providing financial support for the Natural History Study. This clinical research collects the essential data needed to provide our industry partners information about disease progression which guides them as they work to develop treatments and design clinical trials. This effort is paramount to make the necessary advancements in pursuit of a treatment for Ataxia and, ultimately, a cure. 

No funds provided by the National Ataxia Foundation may be used for indirect/F&A costs. 

Print Friendly, PDF & Email
Translate »

Join the Ataxia community today!

Become a free member for exclusive content from NAF.