Clinical Trial Readiness for SCA1 and SCA3 (READISCA)

Purpose

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.

Procedures

  • Blood and cerebrospinal fluid samples
  • Imaging
  • Patient health questionnaires

For study details, please contact Stephen Gullet:

Eligibility 

  • Signed informed consent (no study-related procedures may be performed before the subject has signed the consent form)
  • Subjects of either sex aged 18 to 65 with presence of symptomatic ataxic disease or asymptomatic mutation carrier or
  • Subjects with definite molecular diagnosis of SCA1 or SCA3 or another affected family member
  • Subjects of any age with previous diagnosis of Early stage SCA1 and SCA3
  • Subjects capable of understanding and complying with protocol requirements
  • No changes in physical/occupational therapy status within two months prior to enrollment

Additional criteria apply, please contact Stephen Gullet for more information: 

Age

18 to 65
65 and over

Gender

Male
Female

Can be done from home

No

Keywords

Spinocerebellar Ataxia, Neurology, Ataxia

Principal Investigator

S.H. Subramony, MD

Department

Neurology

Contact Information

stephen.gullett@neurology.ufl.edu

352-273-6003


Be an Informed Participant

Before deciding to participate in a research study, take time to learn about clinical research, how it's conducted and your rights as a research participant. Following are some helpful resources from independent sources. Always remember that a clinical research study is research, not treatment.

Know Who to Contact

  • Eligibility: For questions about a specific study and who is eligible to participate, call or email the contact person listed for that study.
  • Your Rights: For questions about your rights as a research participant, contact the UF Institutional Review Boards at 352-273-9600.
  • Feedback: For general questions or feedback about study listings, email the UF Clinical and Translational Science Institute at CTSI-Studies-L@lists.ufl.edu.

Other Resources

  • HealthStreet: Health-focused services, classes and events, and opportunities to participate in research.
  • ResearchMatch.org: Join a national registry of volunteers willing to be contacted about research studies.

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