With no specific treatment for SCA, this five-year clinical trial readiness grant will establish the world’s largest study participant group so that researchers can collect enough clinical, imaging and biochemical data to design full-scale clinical trials that generate conclusive results on effective treatments. The implications of this readiness study are significant, given that there has been only one pharmaceutical treatment option approved in the last 25 years for ataxia

About SCA

Spinocerebellar ataxia (SCA) is a degenerative disease of the nervous system. It is characterized by degeneration of the brain and spinal cord with progressive clumsiness and difficulty balancing, typically progressing to total disability and resulting in death. The degenerative process mimics that of Huntington’s disease, Parkinson’s disease and amyotrophic lateral sclerosis (ALS). SCA types 1 and 3 are the focus of this clinical trial readiness grant.

SCA1

  • Fastest progressing
  • Shorter clinical trial timelines due to swift progression
  • Sign/symptom onset typically occurs in early adulthood, but can occur between childhood and late adulthood
  • Patients eventually require wheelchair assistance
  • Survival 10-20 years after first symptoms

SCA3

  • Most common SCA in US and Europe, where the study sites are located
  • More prominent worldwide
  • Opportunity for larger number of study participants for clinical trial
  • Onset of signs/symptoms typically occurs in mid-adulthood, but can occur between childhood and late adulthood
  • Patients eventually require wheelchair assistance
  • Survival 10-20 years after first symptoms

Treatment

Current therapies for SCA are limited to supportive therapy. Speech therapy, occupational therapy, and physical therapy are common options, sometimes combined with medication to help manage symptoms. There is no other treatment or cure for SCA.

Aims

This clinical trial readiness grant has three specific aims, with different milestones within each aim that occur over the five-year grant period. The three specific aims are:

Aim 1

Establish the world’s largest participant group of early-stage and symptomless SCA1 and SCA3 individuals

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Aim 2

Validate imaging signs in early stage and symptomless SCA1 and SCA3 individuals

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Aim 3

Adapt recent developments on statistical design and analysis of small population trials to future clinical trials for SCAs

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Study Criteria

Study criteria includes, but is not limited to:

  • A person has a clinical or genetic diagnosis of SCA1 or SCA3
  • A person has a diagnosis of SCA1 or SCA3 in one of his or her first-degree relatives, for example:
    • A person has early ataxia and his or her parent has SCA1 or SCA3
    • A person has no symptoms but tested positive for SCA1 or SCA3
    • A person has no symptoms and has not taken DNA testing, but his or her sibling or parent tested positive for SCA1 or SCA3

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