Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

Description

Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as Machado-Joseph disease and SCA 6). The diseases are rare, slowly progressive, cause increasingly severe neurological difficulties and are variable across and within genotypes. The purpose of this research study is to bring together a group of experts in the field of SCA for the purpose of learning more about the disease. The research questions are: 1. How does your disease progress over time? 2. What are the best ways to measure the progression? 3. Do some genes, other than the gene that is abnormal in your disease, have any effect on the way the disease behaves? This is a nationwide study and we expect that 800 patients will participate all over the USA. The participants will be in the study for an indeterminate period of time. Study visits will be done every 6 or 12 months depending on the participating site.

Conditions

Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6

Study Overview

Study Details

Study overview

Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as Machado-Joseph disease and SCA 6). The diseases are rare, slowly progressive, cause increasingly severe neurological difficulties and are variable across and within genotypes. The purpose of this research study is to bring together a group of experts in the field of SCA for the purpose of learning more about the disease. The research questions are: 1. How does your disease progress over time? 2. What are the best ways to measure the progression? 3. Do some genes, other than the gene that is abnormal in your disease, have any effect on the way the disease behaves? This is a nationwide study and we expect that 800 patients will participate all over the USA. The participants will be in the study for an indeterminate period of time. Study visits will be done every 6 or 12 months depending on the participating site.

Clinical Research Consortium for Spinocerebellar Ataxias (CRC-SCA) to Study Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias (SCA)

Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

Condition
Spinocerebellar Ataxia Type 1
Intervention / Treatment

-

Contacts and Locations

Los Angeles

University of California Los Angeles, Los Angeles, California, United States, 90095

San Francisco

University of California San Francisco, San Francisco, California, United States, 94115

Gainesville

University of Florida, Gainesville, Florida, United States, 32610

Tampa

University of South Florida, Tampa, Florida, United States, 33620

Atlanta

Emory University, Atlanta, Georgia, United States, 30320

Chicago

University of Chicago, Chicago, Illinois, United States, 60637

Baltimore

John Hopkins University, Baltimore, Maryland, United States, 21287

Boston

Harvard University, Boston, Massachusetts, United States, 02114

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55455

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Presence of symptomatic ataxic disease
  • * Definite molecular diagnosis of SCA 1, 2,3,or 6 either in the subject or another affected family member
  • * Willingness to participate in the study and ability to give informed consent.
  • * Age 6 years and above
  • * Known recessive, X-linked and mitochondrial ataxias
  • * Exclusion of SCA 1, 2, 3 and 6 by previous DNA testing,
  • * A lack of willingness to participate in the study

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Florida,

S. Subramony, MD, PRINCIPAL_INVESTIGATOR, University of Florida

Study Record Dates

2024-05-19