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

Conditions

• Heart Failure With Reduced Ejection Fraction

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Lumacaftor 200 MG — DRUG
  Lumacaftor 200 mg q12
• Placebo — DRUG
  Identical placebo

Study Details

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.

Key Dates

First listed

Jul 10, 2026

Start date

Jul 31, 2026

Status verified

Jul 2026

Primary completion

May 31, 2027

Completion

Sep 30, 2027

Study Design

Enrollment

60 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Lumacaftor
  Participants receive 1 tablet of Lumacaftor 200 mg twice daily for 30 days.
• Placebo Comparator: Placebo
  Participants receive 1 tablet of Lumacaftor placebo tablet twice daily for 30 days.

Primary Outcome Measure

Efficacy of lumacaftor treatment in increasing cerebral blood flow versus placebo in HFrEF patients. [ Time Frame: Baseline and 1 month ]

Central Contacts

• Qanatpharma Clinical Program Manager
  1-877-308-4220
  [email protected]

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