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

NCT07252310 | Enrolling By Invitation Phase 4 FDA Drug FDA Device

• 1 other identifier: 25-08029225
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

HFrEF; App; N-of-1; GDMT; StudyU; Beta Blocker

Outcome Measures

Primary Outcomes (3)

• Feasibility of Intervention Measure Score

  The Feasibility of Intervention (FIM) measure is a survey that evaluates an intervention's practicality. Questions are ranked on 1- to 5-point Likert scales, with higher scores indicating higher feasibility and lower indicating less feasibility. Total scores range from 4 to 20. A score of 4 indicates low feasibility and a score of 20 indicates strong feasibility.

  End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

• Intervention Appropriateness Measure Score

  The Intervention Appropriateness Measure (IAM) is a survey that evaluates an intervention's suitability and compatibility. Questions are ranked on 1- to 5-point Likert scales, with higher scores indicating higher appropriateness and lower indicating less appropriateness. Total scores range from 4 to 20. A score of 4 indicates low appropriateness and a score of 20 indicates strong appropriateness.

  End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

• Acceptability of Intervention Measure Score

  Acceptability of Intervention Measure (AIM) is a survey that evaluates how agreeable an intervention is to participants. Questions are ranked on 1- to 5-point Likert scales, with higher scores indicating higher acceptability and lower indicating less acceptability. Total scores range from 4 to 20. A score of 4 indicates low acceptability and a score of 20 indicates strong acceptability.

  End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

Secondary Outcomes (4)

• Change in KCCQ-12 Score

  Baseline, assessed on Day 1. End of Period visit, assessed between Week 3 (minimum) and Week 18 (maximum). End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

• Change in PROMIS-29 Score

  Baseline, assessed on Day 1. End of Period visit, assessed between Week 3 (minimum) and Week 18 (maximum). End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

• Change in PROMIS CF-SF 6a Score

  Baseline, assessed on Day 1. End of Period visit, assessed between Week 3 (minimum) and Week 18 (maximum). End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

• Change in PROMIS Sexual Function Score

  Baseline, assessed on Day 1. End of Period visit, assessed between Week 3 (minimum) and Week 18 (maximum). End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).

Study Arms (1)

GDMT (Beta Blockers)

EXPERIMENTAL

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.

Drug: beta blockers; Device: StudyU Application

Interventions

beta blockers DRUG

Subjects will remain on their current home dose of beta blocker (as prescribed by their treating physician prior to enrollment) during Period 1. In Period 2, the dose will be increased -either doubled or by 50%-to support progression toward the guideline-directed medical therapy (GDMT) target. may choose to continue on the tested dose and conclude the study, or request additional information before selecting their preferred dose. If both the participant and the study clinician agree that further evaluation is needed, a third period will be initiated. In this period, the dose may again be increased-by 50% or doubled relative to the Period 2 dose-if tolerated and agreed upon by the participant. If the new dose is well-tolerated and the participant agrees to continue with dose escalation after reviewing their data, the dose will be increased again-either doubled or increased by 50%-for the next period (Period 3). The study team will continue to collect data.

GDMT (Beta Blockers)

StudyU Application DEVICE

StudyU is a novel mobile app developed for the design of and conduct of N-of-1 trials. StudyU consists of the StudyU Designer, a web platform and mobile app, accessible through the website https://designer.studyu.health; the StudyU App for smartphones, which can be downloaded from Apple and Google app stores, and the secure backend where data is stored. All components of StudyU were developed by Dr. Stefan Konigorski and his colleagues at Hasso-Plattner Institute (HPI) for Digital Engineering at the University of Potsdam. The StudyU platform allows investigators to easily operationalize their study processes in the StudyU designer. In the StudyU designer, investigators indicate at which frequency subjects will complete assessments. For this pilot trial, a WCM-specific instance of StudyU will be developed and hosted on the secure WCM server. Only the WCM study team will have access to the subject data from the StudyU App.

GDMT (Beta Blockers)

Eligibility Criteria

• Age: 65 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• Adults ≥ 65 years old
• 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
• Taking less than the maximal dose of beta blocker per physician recommendation at time of enrollment
• 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

You may not qualify if:

• Clinical instability (this N-of-trial is appropriate for stable conditions only)
• Decompensated HF
• Hospitalized in the past 30 days
• Medication changes or procedures in prior 14 days (to prevent confounding from other interventions) at PI discretion
• Do not have access to a smartphone or tablet
• Estimated life expectancy \<6 months
• Moderate-severe dementia or psychiatric disorder precluding informed consent
• Language barrier that will preclude informed consent and ability to comprehend study procedures
• History of noncompliance or inability to complete study procedures
• Enrollment in a clinical trial not approved for co-enrollment
• Any condition that, in Principal Investigator or treating physician's opinion, makes the patient unsuitable for study participation

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead

Study Sites (1)

Weill Cornell Medical College

New York, New York, 10021, United States

Location

Related Publications (5)

• Goyal P, Safford MM, Hilmer SN, Steinman MA, Matlock DD, Maurer MS, Lachs MS, Kronish IM. N-of-1 trials to facilitate evidence-based deprescribing: Rationale and case study. Br J Clin Pharmacol. 2022 Oct;88(10):4460-4473. doi: 10.1111/bcp.15442. Epub 2022 Jul 13.

  PMID: 35705532 BACKGROUND

• Shiffman S, Stone AA, Hufford MR. Ecological momentary assessment. Annu Rev Clin Psychol. 2008;4:1-32. doi: 10.1146/annurev.clinpsy.3.022806.091415.

  PMID: 18509902 BACKGROUND

• Heidenreich PA, Bozkurt B, Aguilar D, Allen LA, Byun JJ, Colvin MM, Deswal A, Drazner MH, Dunlay SM, Evers LR, Fang JC, Fedson SE, Fonarow GC, Hayek SS, Hernandez AF, Khazanie P, Kittleson MM, Lee CS, Link MS, Milano CA, Nnacheta LC, Sandhu AT, Stevenson LW, Vardeny O, Vest AR, Yancy CW; ACC/AHA Joint Committee Members. 2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure: A Report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. Circulation. 2022 May 3;145(18):e895-e1032. doi: 10.1161/CIR.0000000000001063. Epub 2022 Apr 1.

  PMID: 35363499 BACKGROUND

• Verhestraeten C, Heggermont WA, Maris M. Clinical inertia in the treatment of heart failure: a major issue to tackle. Heart Fail Rev. 2021 Nov;26(6):1359-1370. doi: 10.1007/s10741-020-09979-z.

  PMID: 32474794 BACKGROUND

• Rao VN, Greene SJ. Breaking Clinical Inertia in Heart Failure Management. Jt Comm J Qual Patient Saf. 2022 Jan;48(1):3-4. doi: 10.1016/j.jcjq.2021.11.002. Epub 2021 Nov 11. No abstract available.

  PMID: 34840128 BACKGROUND

MeSH Terms

Conditions

Heart Failure; Heart Failure, Systolic; Heart Diseases; Alzheimer Disease

Interventions

Adrenergic beta-Antagonists

Condition Hierarchy (Ancestors)

Cardiovascular Diseases; Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Physiological Effects of Drugs

Study Officials

• Parag Goyal, MD, MSc

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2025
• First Posted: November 26, 2025
• Study Start: February 10, 2026
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): August 1, 2027
• Last Updated: February 19, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)