Home-Reported Outcomes in People With Cystic Fibrosis Taking Highly Effective CFTR Modulator Therapy
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04798014 |
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Recruitment Status :
Recruiting
First Posted : March 15, 2021
Last Update Posted : September 17, 2021
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| Condition or disease |
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| Cystic Fibrosis |
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.
| 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 |
- 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.
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.
| 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 |
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.
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
| Contact: Cynthia Brown, MD | (317) 278-0175 | cyndbrow@iu.edu | |
| Contact: Clement Ren, MD | 215-590-3749 | renc@chop.edu |
| 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 | |
| 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: | September 17, 2021 |
| Last Verified: | September 2021 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Trikafta Triple Combination Therapy Folia Health |
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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 |