Clinical Trial Readiness for SCA1 and SCA3

Summary

The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-European collaborations, the investigators will establish the world's largest cohorts of subjects at the earliest disease stages, who will benefit most from treatments, validate an ability to detect disease onset and early progression by imaging markers, even prior to ataxia onset, and identify clinical trial designs that will generate the most conclusive results on treatment efficacy with small populations of patients.

Conditions

• Spinocerebellar Ataxia Type 1
• Spinocerebellar Ataxia 3

Sponsors & Collaborators

• University of Michigan

  collaborator OTHER

• University of Minnesota

  collaborator OTHER

• University of Utah

  collaborator OTHER

• University of California, Los Angeles

  collaborator OTHER

• University of Chicago

  collaborator OTHER

• University of South Florida

  collaborator OTHER

• Harvard University

  collaborator OTHER

• Johns Hopkins University

  collaborator OTHER

• University of Florida

  collaborator OTHER

• Columbia University

  collaborator OTHER

• Emory University

  collaborator OTHER

• University of California, San Francisco

  collaborator OTHER

• University of Alabama at Birmingham

  collaborator OTHER

• University of Colorado, Denver

  collaborator OTHER

• University of Rochester

  collaborator OTHER

• Stanford University

  collaborator OTHER

• Northwestern University

  collaborator OTHER

• German Center for Neurodegenerative Diseases (DZNE)

  collaborator OTHER

• Institut de Recherche sur la Moelle épinière et l'Encéphale

  collaborator OTHER

• University of Pennsylvania

  collaborator OTHER

• Ohio State University

  collaborator OTHER

• University of Iowa

  collaborator OTHER

• The Methodist Hospital Research Institute

  lead OTHER

Principal Investigators

• Tetsuo Ashizawa, MD · The Methodist Hospital Research Institute

• Hank Paulson, MD, PhD · University of Michigan

• Gulin Oz, MD · University of Minnesota

• Thomas Klockgether, MD · University Hospital Bonn - DZNE

• Alexandra Durr, MD, PhD · Hôpital Universitaire Pitié-Salpêtrière - ICM/SPATAX

• Sheng Han Kuo, MD · Columbia University

• George Wilmot, MD, PhD · Emory University

• Liana Rosenthal, MD · Johns Hopkins University

• Chiadikaobi Onyike, MD · Johns Hopkins University

• Puneet Opal, MD, PhD · Northwestern University

• Sharon Sha · Stanford University

• Talene Yacoubian, MD, PhD · University of Alabama at Birmingham

• Susan Perlman, MD · University of California, Los Angeles

• Michael Geschwind, MD,PhD · University of California, San Francisco

• Trevor Hawkins, MD · University of Colorado, Denver

• Christopher Gomez, MD, PhD · University of Chicago

• SH Subramony, MD · University of Florida

• Vikram Shakkottai, MD, PhD · University of Texas

• Khalaf Bushara, MD · University of Minnesota

• Theresa Zesiewicz, MD · University of South Florida

• Stefan Pulst, MD, PhD · University of Utah

• Jeremy Schmahmann, MD, PhD · Harvard University

• Peter Barker, MD · Johns Hopkins University

• Haris I Sair, MD · Johns Hopkins University

• Veronica Santini, MD · Stanford University

• Eva-Maria Ratai, MD · Harvard University

• Thomas Mareci, MD · Universtiy of Florida, Gainesville

• Laura Scorr, MD · Emory University

• Peggy C Nopoulos, MD · University of Iowa

• Ali G Hamedani, MD, PhD · University of Pennsylvania

• Yaz Y Kisanuki, MD, FAAN · Ohio State University

• Peter Morrison, DO · University of Rochester

Eligibility

Min Age

18 Years

Max Age

65 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2018-08-16

Primary Completion

2023-12-31

Completion

2023-12-31

Countries

• United States

Study Locations