Status:
COMPLETED
Integrated Functional Evaluation of the Cerebellum
Lead Sponsor:
Institut National de la Santé Et de la Recherche Médicale, France
Collaborating Sponsors:
Biogen
Ionis Pharmaceuticals, Inc.
Conditions:
Spinocerebellar Ataxia Type 2
Spinocerebellar Ataxia Type 7
Eligibility:
All Genders
18+ years
Brief Summary
One of the main objectives of this project is to validate potential biological, clinical and/or imaging biomarkers in SCA patients through a multimodal assessment, for future ASOs trials.
Detailed Description
Spinocerebellar ataxias (SCAs) are autosomal dominantly inherited neurological disorders, characterized by a predominant atrophy of the cerebellum and the brainstem. The most common forms are caused b...
Eligibility Criteria
Inclusion
- Common inclusion criteria for all participants:
- Ability to walk independently 30 foot without an assistive device
- Able to stand unassisted for 30 seconds
- Affiliated with the French social security, or a social security equivalent, if they are not French.
- Capacity to consent
- Signed Informed Consent by the subject
- Ability to undergo MRI scanning
- Inclusion criteria for SCA patients:
- Genetic diagnosis of SCA 2 or 7 (available CAG repeat length)
- SARA score ≤15
- Inclusion criteria for control participants:
- Negative Genetic diagnosis of SCA2/SCA7 available
- No significant neurological symptoms
- SARA score \< 5
- Common inclusion criteria for elective participant for CSF sampling:
- • Ability to undergo a lumbar puncture
- Exclusion criteria
- Subjects currently receiving, or having received within 2 months prior to enrolment into this study, any investigational drug
- Pregnancy or breastfeeding
- Genotype consistent with other inherited ataxias
- Changes in coordinative physical and occupational therapy for ataxia 2 months prior to study participation
- Concomitant disorder(s) or condition(s) that affects assessment of ataxia or severity of ataxia during this study
- Contra-indications to MRI examination
- Person deprived of their liberty by judicial or administrative decision
Exclusion
Key Trial Info
Start Date :
May 28 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2022
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT04288128
Start Date
May 28 2020
End Date
June 1 2022
Last Update
November 18 2025
Active Locations (1)
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1
Institut du Cerveau - Paris Brain Institute
Paris, France, 75013