Cystic FibrosisNCT04798014
HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy
An observational study of Cystic Fibrosis, run by Indiana University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 860 participants
- Where
- 1 siteUnited States
- Study dates
- 26 May 2021to 15 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University School of Medicine
Indianapolis, Indiana, 46202
About this study
Official title: Home-Reported Outcomes in People With Cystic Fibrosis (CF) Taking Highly Effective Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy
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.
Read the detailed description
This study will be a prospective, observational cohort study to investigate the changes in lung function among individuals who have made treatment changes after initiating treatment with elexacaftor/tezacaftor/ivacaftor (ETI). Data will be collected from Folia Health, and the Cystic Fibrosis Foundation Patient Registry (CFFPR) for participants who are registered. Participation in the study will occur through the Folia application where study participants will be directed to a specific HERO-2 study dashboard. Individuals (n=860) taking ETI may be enrolled at participating sites, through social media recruitment or from existing users of Folia Health. 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 CF patient-reported outcome (PRO) questionnaires. Participants will also be asked to self-report instances of changes to their treatment plan, and pulmonary exacerbations. Data collected within Folia will be linked to the CFFPR for individual outcomes with regards to clinical data including anthropometric measures, lung function, complications, and IV-treated pulmonary exacerbations. This study will not provide or recommend any treatment recommendations. All direction for medication usage is solely at the discretion of the patient's physician in accordance with their usual practice.
The primary objective for the study will be to evaluate changes in lung function as measured by forced expiratory volume over one second (FEV1) percent predicted in individuals who report making at least one change to chronic daily respiratory therapies upon study entry and during the 12 month follow up compared to those who do not make changes to chronic daily therapies. Secondary endpoints will include a measurement of the proportion of individuals making changes to chronic daily therapy, the need to resume chronic daily therapy, health-related quality of life, symptom severity and symptom frequency, and pulmonary exacerbations requiring IV antibiotics.
- Conditions
- Cystic Fibrosis
- Keywords
- Trikafta, Triple Combination Therapy, Folia Health
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in FEV1 percent predicted
The primary outcome measure of this study is change in FEV1 percent predicted at 12 months.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2021
- Study start26 May 2021
- Primary completion15 Aug 2023
- Study completion15 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 860 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Collaborators
- Folia Health, Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Cynthia Brown, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04798014
- Sponsor's protocol ID
- 11375
- Status last verified
- Jul 2026
- First submitted
- 11 Mar 2021
- Last updated
- 28 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Rosen BH, Psoter KJ, Sabadosa KA, Hergenroeder GE, Bendy LL, Luo NM, Zhang C, Ren CL, Brown CD. Using Real-World Research to Study the Impact of Chronic Daily Therapy Discontinuation in Cystic Fibrosis: The Home-Reported Outcomes in Cystic Fibrosis 2 Study Design. CHEST Pulm. 2024 Jun 29;2(4):100080. doi: 10.1016/j.chpulm.2024.100080. eCollection 2024 Dec.
PubMed 42548438
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Home-Reported Outcomes in People With Cystic Fibrosis (CF) Taking Highly Effective Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy
- ClinicalTrials.gov ID
- NCT04798014
- Acronym / sponsor ID
- 11375
- Conditions
- Cystic Fibrosis
- Keywords
- Trikafta, Triple Combination Therapy, Folia Health
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 860 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Collaborators
- Folia Health, Cystic Fibrosis Foundation
- Study start
- 26 May 2021 (actual)
- Primary completion
- 15 Aug 2023 (actual)
- Study completion
- 15 Aug 2023 (actual)
- First posted
- 15 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study will be a prospective, observational cohort study to investigate the changes in lung function among individuals who have made treatment changes after initiating treatment with elexacaftor/tezacaftor/ivacaftor (ETI). Data will be collected from Folia Health, and the Cystic Fibrosis Foundation Patient Registry (CFFPR) for participants who are registered. Participation in the study will occur through the Folia application where study participants will be directed to a specific HERO-2 study dashboard. Individuals (n=860) taking ETI may be enrolled at participating sites, through social media recruitment or from existing users of Folia Health. 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 CF patient-reported outcome (PRO) questionnaires. Participants will also be asked to self-report instances of changes to their treatment plan, and pulmonary exacerbations. Data collected within Folia will be linked to the CFFPR for individual outcomes with regards to clinical data including anthropometric measures, lung function, complications, and IV-treated pulmonary exacerbations. This study will not provide or recommend any treatment recommendations. All direction for medication usage is solely at the discretion of the patient's physician in accordance with their usual practice.
The primary objective for the study will be to evaluate changes in lung function as measured by forced expiratory volume over one second (FEV1) percent predicted in individuals who report making at least one change to chronic daily respiratory therapies upon study entry and during the 12 month follow up compared to those who do not make changes to chronic daily therapies. Secondary endpoints will include a measurement of the proportion of individuals making changes to chronic daily therapy, the need to resume chronic daily therapy, health-related quality of life, symptom severity and symptom frequency, and pulmonary exacerbations requiring IV antibiotics.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in FEV1 percent predicted
Time frame: 12 months
The primary outcome measure of this study is change in FEV1 percent predicted at 12 months.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2026.
- Derived from this study: PubMed 42548438Rosen BH, Psoter KJ, Sabadosa KA, Hergenroeder GE, Bendy LL, Luo NM, Zhang C, Ren CL, Brown CD. Using Real-World Research to Study the Impact of Chronic Daily Therapy Discontinuation in Cystic Fibrosis: The Home-Reported Outcomes in Cystic Fibrosis 2 Study Design. CHEST Pulm. 2024 Jun 29;2(4):100080. doi: 10.1016/j.chpulm.2024.100080. eCollection 2024 Dec.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Indiana University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in FEV1 percent predicted (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04798014, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2026.