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

Official title

Pilot Study of Acceptability and Feasibility of StudyU for N-of-1 Trials

Overview

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.

Detailed 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.

Interventions

• Drug beta blockers
  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
• Device StudyU Application
  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

Primary outcome measures

• Feasibility of Intervention Measure Score [Time frame: End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).]
• Intervention Appropriateness Measure Score [Time frame: End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).]
• Acceptability of Intervention Measure Score [Time frame: End of intervention visit, assessed between Week 6 (minimum) and Week 18 (maximum).]

Secondary outcome measures (4)

• Change in KCCQ-12 Score [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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).]

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Other

Study locations

United States · 1 center

• Weill Cornell Medical College — New York

Publications

• 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
• 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
• 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 t PMID 35363499
• 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
• 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

Identifiers

NCT: NCT07252310 · 25-08029225

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗