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Home-Reported Outcomes in People With Cystic Fibrosis Taking Highly Effective CFTR Modulator Therapy

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ClinicalTrials.gov Identifier: NCT04798014
Recruitment Status : Recruiting
First Posted : March 15, 2021
Last Update Posted : June 3, 2021
Sponsor:
Collaborators:
Indiana University School of Medicine
Folia Health
Information provided by (Responsible Party):
Cystic Fibrosis Foundation

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.

Condition or disease
Cystic Fibrosis

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 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.

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Study Type : Observational
Estimated Enrollment : 860 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Home-Reported Outcomes in People With Cystic Fibrosis Taking Highly Effective CFTR Modulator Therapy
Actual Study Start Date : May 26, 2021
Estimated Primary Completion Date : December 1, 2022
Estimated Study Completion Date : December 1, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis




Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04798014


Contacts
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Contact: Cynthia Brown, MD (317) 278-0175 cyndbrow@iu.edu
Contact: Clement Ren, MD clren@iu.edu

Locations
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United States, Indiana
Indiana University School of Medicine Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Cynthia Brown, MD       cyndbrow@iu.edu   
Contact: Lisa Bendy       lbendy@iu.edu   
Sponsors and Collaborators
Cystic Fibrosis Foundation
Indiana University School of Medicine
Folia Health
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Responsible Party: Cystic Fibrosis Foundation
ClinicalTrials.gov Identifier: NCT04798014    
Other Study ID Numbers: HERO-2
First Posted: March 15, 2021    Key Record Dates
Last Update Posted: June 3, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cystic Fibrosis Foundation:
Trikafta
Triple Combination Therapy
Folia Health
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases