Lumacaftor Yields Reversal of Impaired Cerebral Blood Flow in Heart Failure Patients

Cognitive impairment (CI) is highly prevalent in patients with heart failure with reduced ejection fraction (HFrEF), which has significant implications for disease management, quality of life and clinical outcomes. Currently, there are no specific treatments for CI aside from the current standard of care therapy for HF, making this a high unmet medical need. Impaired cerebral autoregulation is a proposed mechanistic factor that leads to cerebral hypoperfusion, ischemic damage and the development for CI. Preclinical data indicates that restoring CFTR-protein expression normalizes cerebral microvascular function and cerebral blood flow (CBF) in models of HF. The purpose of this study is to investigate whether CFTR-targeting therapy enhances cerebral perfusion and cognitive function in heart failure patients using the CFTR-corrector Lumacaftor.

NCT Number	NCT07695090
Lead Sponsor	Qanatpharma AG Collaborators: Qanatpharma Canada LTD
Conditions	Heart Failure With Reduced Ejection Fraction
Enrollment	60 participants
Start Date	2026-07
Primary Completion	2027-05 (estimated)
Study Completion	2027-09 (estimated)
Updated on ClinicalTrials.gov	2026-07-17