HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy

NCT04798014 | Completed

• 1 other identifier: 11375
• Study Type: observational
• Target: 860
• Locations: 1 country
• Sites: 1

Brief Summary

This is an observational cohort study, using data from Folia Health and the Cystic Fibrosis Foundation Patient Registry (CFFPR). Individuals taking elexacaftor/tezacaftor/ivacaftor (ETI) may be enrolled through the Folia application. During the 12-month study period, participants will be asked to track their routine treatment and medication usage, daily symptoms, and monthly review with validated patient-reported outcome (PRO) questionnaires. Participants will also be asked to self-report instances of changes to their treatment plan, and pulmonary exacerbations. There are no study-associated site visits.

Conditions

Cystic Fibrosis

Keywords

Trikafta; Triple Combination Therapy; Folia Health

Outcome Measures

Primary Outcomes (1)

• Change in FEV1 percent predicted

  The primary outcome measure of this study is change in FEV1 percent predicted at 12 months.

  12 months

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals diagnosed with Cystic Fibrosis, and who are on ETI through an FDA-labeled indication are eligible for this study. Study participants will be using Folia Health, a web-based application to track daily care and must have access to a mobile phone or computer with an internet connection.

You may qualify if:

• Written informed consent (and assent when applicable) obtained from subject or subject's legal guardian.
• Is willing and able to adhere to protocol requirements.
• ≥ 12 years of age at enrollment.
• Diagnosed with CF.
• Prescribed ETI for an on-label indication.
• Participant has access to a mobile phone or tablet (iPhone or Android platform) or computer.
• Participant has access to an internet connection.

You may not qualify if:

• Participant is enrolled in the randomized portion of the SIMPLIFY study (NCT04378153).
• Participant does not have access to the necessary technology.
• Participant is a recipient of a solid organ transplant (i.e. lung, liver, pancreas, kidney).
• Participant cannot communicate in English.

Sponsors & Collaborators

• Indiana University: lead
• Folia Health: collaborator
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Clinical Medicine

Study Record Dates

• First Submitted: March 11, 2021
• First Posted: March 15, 2021
• Study Start: May 26, 2021
• Primary Completion: August 15, 2023
• Study Completion: August 15, 2023
• Last Updated: July 28, 2026

Record last verified: 2026-07

Locations

US (1)