Pilot Study of StudyU for N-of-1 Trials in HFrEF Patients (N-of-1 App)

Weill Cornell Medicine (WCM)

Status and phase: Invitation-only; Phase 4

Conditions: Heart Failure, Systolic; HFrEF - Heart Failure With Reduced Ejection Fraction; Heart Failure; Heart Diseases

Treatments: Device: StudyU Application; Drug: beta blockers

Study type: Interventional

Funder types: Other

Identifiers

NCT07252310

25-08029225

Details and patient eligibility

About

This study will look at whether using a phone app called StudyU can help people with Heart Failure with Reduced Ejection Fraction (HFrEF) reach their recommended dose on their beta blocker.

Full description

The overarching goal of this study is to determine feasibility of the StudyU app in N-of-1 trials. The study intervention is N-of-1 trials, facilitated by a mobile-based application, StudyU. The study team will utilize a single-arm sequential design N-of-1 study in which subjects who are not yet at the GDMT for beta blockers will test multiple doses of GDMT to understand the maximally tolerated dose using an N-of-1 trial format. This is similar to an approach used in a previous study, Pilot Deprescribing N-of-1 Trials for beta blockers in HFpEF (NCT04757584) but will test the addition of a mobile-based app to collect vital signs, monitor side effects, and monitor patient-reported outcomes.

Enrollment: 5 estimated patients

Sex: All

Ages: 65+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Adults ≥ 65 years old
2. A history of HFrEF per electronic medical record review/clinician impression that is defined as an EF <50% that is shown from any imaging modality
3. Taking less than the maximal dose of beta blocker per physician recommendation at time of enrollment
4. Access to a smartphone or device that can perform many of the same functions as a computer, typically having a touchscreen interface, internet access, and an operating system capable of running downloaded applications

Exclusion criteria

1. Clinical instability (this N-of-trial is appropriate for stable conditions only)

   1. Decompensated HF
   2. Hospitalized in the past 30 days
   3. Medication changes or procedures in prior 14 days (to prevent confounding from other interventions) at PI discretion

2. Do not have access to a smartphone or tablet

3. Estimated life expectancy <6 months

4. Moderate-severe dementia or psychiatric disorder precluding informed consent

5. Language barrier that will preclude informed consent and ability to comprehend study procedures

6. History of noncompliance or inability to complete study procedures

7. Enrollment in a clinical trial not approved for co-enrollment

8. Any condition that, in Principal Investigator or treating physician's opinion, makes the patient unsuitable for study participation

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

GDMT (Beta Blockers)

Experimental group

Description:

This study consists of a single arm. Subjects will complete study activities remotely. Following enrollment, daily assessments will be conducted using the StudyU mobile application. Additional assessments will occur at the end of each study period, completed by phone, WCM Zoom, or via email, depending on subject preference. The study involves testing multiple doses of each subject's guideline-directed medical therapy (GDMT), specifically beta-blockers. Doses will include the subject's current home dose (as prescribed by their treating physician prior to enrollment), as well as one or more increased -and, if needed, decreased-doses that are near or at the guideline-directed maximum. All dosing decisions will be made collaboratively by the PI, the subject's clinician, and the participant, taking into account titration guidelines, physician guidance, and subject symptoms and preferences. Each subject will complete a minimum of two periods. The number of periods is adaptive.

Treatment:

Drug: beta blockers

Device: StudyU Application