Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)

NCT ID: NCT06472557

Last Updated: 2025-07-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-06-04

Study Completion Date

2028-12-31

Brief Summary

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This international, multi-center, multi-modal, and prospective observational cohort study aims to validate trial outcomes for capturing disease progression in Spinocerebellar Ataxia Type 27B (SCA27B), with combined multi-modal capture of clinical outcome assessments, digital-motor assessments, and molecular biomarkers.

Detailed Description

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The investigators will perform an international, multi-center, multi-modal, and registry-based standardized prospective Natural History Study (NHS) in Spinocerebellar Ataxia Type 27B (SCA27B), including the presymptomatic phase of the disease (i.e. presymptomatic subjects at risk for SCA27B). Participants will be assessed annually. Clinical data, including clinician-reported outcomes and patient-focused outcomes, will be entered into a clinical database customized to the requirements of this specific study (SCA27B Registry; www.ataxia-registries.org). Digital-motor outcomes comprise digital gait assessment by wearable sensors, and digital assessment of upper limb movements by Q-Motor. At all study visits, participants will be asked to donate biosamples; and participants can elect to participate in sampling of blood, urine, CSF, and/or a skin biopsy. Based on this multimodal protocol, the study aims to determine the most sensitive, comprehensive, and reliable outcome measures for future therapeutic trials in SCA27B.

Conditions

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Spinocerebellar Ataxia Type 27B

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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SCA27B

Participants with genetically confirmed SCA27B (OMIM 620174), i.e. ≥250 uninterrupted GAA repeat expansions in FGF14, will be recruited. Target sample size for the SCA27B cohort is 200 participants.

No interventions assigned to this group

SCA27B risk subjects

First-degree relatives at risk for SCA27B, both with known and unknown carrier status, will be included to capture presymptomatic disease stages. Target sample size for the risk cohort is 50 participants

No interventions assigned to this group

Unrelated healthy controls

Unrelated healthy controls may undergo the same study procedures as the SCA27B cohort to determine age-related effects on the multimodal outcomes. Target sample size for the control cohort is 50.

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* SCA27B: genetic diagnosis of ≥250 uninterrupted GAA repeat expansions in FGF14
* SCA27B risk subject: asymptomatic first-degree relative of SCA27B participant with known or unknown carrier status
* Unrelated healthy controls: no signs or history of neurological or psychiatric disease AND
* Written informed consent AND
* Participants are willing and able to comply with study procedures

Exclusion Criteria

* SCA27B: Missing informed consent
* SCA27B risk subjects: Missing informed consent
* Unrelated healthy controls: Missing informed consent, or concurrent neurological, orthopedic, or other diseases interfering with the motor assessments
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University Hospital Tuebingen

OTHER

Sponsor Role lead

Responsible Party

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Prof. Dr. Matthis Synofzik

Principal Investigator, Leading Consultant

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Matthis Synofzik, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

University Hospital Tübingen

Andreas Traschütz, Dr. Dr.

Role: PRINCIPAL_INVESTIGATOR

University Hospital Tübingen

Locations

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Department of Neurology, Motol University Hospital, Second Faculty of Medicine, Charles University

Prague, , Czechia

Site Status RECRUITING

Center for Neurology & Hertie-Institute for Clinical Brain Research, Dept. for Neurodegenerative Diseases

Tübingen, Baden-Wurttemberg, Germany

Site Status RECRUITING

Department of Neurology & Center for Translational Neuro- and Behavioral Sciences, Essen University Hospital, University of Duisburg-Essen

Essen, North Rhine-Westphalia, Germany

Site Status RECRUITING

Department of Neurology, Donostia University Hospital, BioGipuzkoa Health Research Institute

San Sebastián, Gipuzkoa, Spain

Site Status RECRUITING

Countries

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Czechia Germany Spain

Central Contacts

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Matthis Synofzik, Prof. Dr.

Role: CONTACT

+49 7071 29 ext. 82060

Andreas Traschütz, Dr. Dr.

Role: CONTACT

+49 7071 29 ext. 61890

Facility Contacts

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Olga Brýksiová

Role: primary

+420 702 269 277

Lisa Graf, MSc

Role: primary

+49 7071 29 ext. 85374

Friedrich Erdlenbruch, Dr.

Role: primary

+49 201 723 ext. 2180

Ioana Croitoru, SC

Role: primary

+34 94300 ext. 7027

Other Identifiers

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SCA27B-NHS

Identifier Type: -

Identifier Source: org_study_id

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