None listed
Conditions
Brief summary
This is a feasibility study to see whether Pregabalin decreases the severity and/or frequency of coughing associated with CANVAS syndrome. This study is a randomised, double blinded, placebo controlled cross over study, which means that all participants will receive both pregabalin and a placebo at different stages in the study but neither the participants nor the study team will know who is on placebo or pregabalin, To determine if this drug has any effect we will monitor the frequency of coughing by a smartphone app and the severity of the symptoms through surveys and interviews.
Interventions
This study is divided into a two week Screening Phase, followed by 12 weeks of either placebo or pregabalin (treatment phase one) followed by a four week washout period where no drug/placebo is administered, followed by another 12 week period of either pregabalin or placebo (treatment phase two). At the end of 12 weeks there is a final safety period of four weeks (End Phase) were no drug/placebo is administered but the study team continues to follow the participants.
Participants will receive pr
This study is divided into a two week Screening Phase, followed by 12 weeks of either placebo or pregabalin (treatment phase one) followed by a four week washout period where no drug/placebo is administered, followed by another 12 week period of either pregabalin or placebo (treatment phase two). At the end of 12 weeks there is a final safety period of four weeks (End Phase) were no drug/placebo is administered but the study team continues to follow the participants. Participants will receive pregabalin, as an oral capsule at 75 mg twice per day for 12 weeks within a 36 week cross over study. Unused tablets will be returned to dispenser for counting to monitor compliance. For participants who must reduce their dosage following intolerable adverse drug reaction, they will reduce to one capsule per day. For those who still experience unmanaged cough, they may increase their dosage. This will be increased to three capsules per day if they are tolerating the medication and their blood test results are not deranged. Participants may be offered this increase or decrease of dosage at the end of the first week of each treatment phase. Safety bloods are to be performed at screening, four weeks into treatment phase one and four weeks in treatment phase two. Additional safety bloods may be ordered in the medical opinion of the PI.
Sponsors
Richard Roxburgh
Study design
Allocation
Randomised controlled trial
Intervention model
Crossover
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Healthy volunteers
No
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
Outcome results
None listed