Pregabalin for the treatment of Cerebellar Ataxia with Neuropathy and Vestibular Areflexia Syndrome (CANVAS) associated chronic cough.

Phase 2

• Conditions: Cerebellar Ataxia with Neuropathy and Vestibular Areflexia Syndrome (CANVAS); chronic cough; Neurological - Neurodegenerative diseases

• Registration Number: ACTRN12623001356651
• Lead Sponsor: Richard Roxburgh

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-12-21
• Last Update Posted: 8 January 2024

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

•Participants with neurological symptoms attributable to RFC1 pathology (neuropathy, vestibular failure, ataxia)
•Positive RFC1 genetic test (either biallelic pathological expansion or pathological expansion and pathological variant).
•> 1 year of chronic cough
•18 years and over
•Can give informed consent.
•Has access to a smart phone.

Exclusion Criteria

•History of cancer (other than skin squamous cell carcinoma (SSC) or basal cell carcinoma (BCC)).
•History of severe renal impairment (glomerular filtration rate (GFR) < 30)
•History of intolerance to pregabalin
•Pregnancy*/breastfeeding.
•Active respiratory disease.
•Current or recently quit (< 6 months) smokers.
•Angiotensin Converting Enzyme (ACE) inhibitor use.
•Productive cough.
•Use of any pregabalin, gabapentin within 3 months of baseline visit
•Comorbid medical condition which, in the opinion of the Principal Investigator (PI) will either confound the outcome of the study, or place the participant at risk
•Blood test abnormalities at screening indicating severe liver or kidney dysfunction

Study & Design

• Study Type: Interventional
• Study Design: Not specified