Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| STRC-113-20-01 | Other Grant/Funding Number | Cystic Fibrosis Foundation |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Johns Hopkins University | OTHER |
Not provided
Not provided
Not provided
Not provided
This qualitative longitudinal study is designed to elicit and thematically analyze the perspectives of SIMPLIFY subjects about treatment withdrawal research and treatment burden in the context of triple combination CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapy (TCT). Cohorts will be determined based on sub-groups of the assigned treatment arms from the SIMPLIFY study. Participants will be asked to participate in two interviews; the first interview will be conducted at the completion of the SIMPLIFY study and the second interview will be conducted approximately 4 months after the first interview. Trained research staff will conduct the semi-structured interviews that will be audio-recorded and transcribed for thematic analysis. Each interview should last between 30-60 minutes.
The recommended daily treatment regimen for CF is complex and results in high treatment burden for many people with CF (PwCF). Newly developed triple combination CFTR modulator therapy (TCT) has shown dramatic health benefits on top of standard CF therapies in a pivotal clinical trial and will soon be a treatment option for a larger population of PwCF. Widespread implementation of TCT will provide an unprecedented opportunity to rigorously study whether PwCF can safely discontinue some previously prescribed daily therapies. The randomized, open-label SIMPLIFY study has been designed to test the hypothesis that withdrawal of dornase alpha and/or nebulized hypertonic saline (HS) is not inferior to continuation of one or both treatments with respect to short-term change in lung function. As this is the first of its kind study design in CF, it unknown how people with CF thinking about withdrawal studies or discontinuing treatments.
Here, the investigators intend to conduct a qualitative interview study that complements the SIMPLIFY protocol in which the investigators aim to assess the experiences of PwCF participating in a treatment withdrawal study. In particular, the investigators will examine post-hoc the beliefs and opinions of a representative subgroup of SIMPLIFY participants about treatment withdrawal research and perceived treatment burden. This qualitative study will provide the opportunity to learn about behaviors after the SIMPLIFY trial, including restarting vs. continuing to maintain medications, withdrawing other medications or therapies, and how patients and families partner with care teams around these decisions. Through these interviews, the investigators will learn how individuals with CF perceive taking part in a drug withdrawal study and how participants discern treatment assignment.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group A | subjects in the hypertonic saline study randomized to discontinue or maintain therapy |
| |
| Group B | subjects in the dornase alfa study randomized to discontinue or maintain therapy |
| |
| Group C | subjects who were randomized but withdrew early from the SIMPLIFY Study. |
| |
| Group D | subjects in the hypertonic saline study randomized to discontinue or maintain therapy with FEV1% predicted between 40 and <60% |
| |
| Group E | caregiver participants (parents and legal guardians of eligible patient participants less than 18 years of age who were randomized in the SIMPLIFY study) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Qualitative Interview | Behavioral | 2 Semi-Structured Qualitative Interviews |
|
| Measure | Description | Time Frame |
|---|---|---|
| Interview #1 | Questions to evaluate (A) attitudes and beliefs regarding participation in a withdrawal study and (B) perceptions of treatment burden in the context of having participated in a withdrawal study. | to be conducted at the time of enrollment (post SIMPLIFY study) |
| Interview #2 | Questions to evaluate pwCF's perceptions of (A) factors that affected the decision to use or not use dornase alfa and/or hypertonic saline post-trial participation and satisfaction with the decision and (B) factors that would affect decision-making about whether to withdraw treatments in the future. | to be conducted 4 months following the date of the first interview |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Participant's Perceptions in Interviews | To determine if perceptions about treatment burden and withdrawal study participation vary by the parent study treatment arm assignment (e.g. randomized to continue or stop dornase alfa and/or hypertonic saline). | from day one to approximately month 8 |
| Change in Participant's Decision-making in Interviews |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Participant Perceptions | To determine if individuals who did not maintain treatment assignment (i.e., person randomized to discontinue treatment restarted the treatment OR person randomized to maintain treatment discontinued it) have different perceptions about treatment burden | from day one to approximately month 8 |
Inclusion Criteria:
Patient Participant:
Caregiver Participant:
Exclusion Criteria:
An individual who meets any of the following criteria will be excluded from participation in this study:
1. Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data
Not provided
Not provided
Not provided
Patient participants will consist of male and female Cystic Fibrosis patients ages 14 and older who were randomized in the SIMPLIFY study. QUEST researchers will focus on assuring a balanced distribution across sub-groups (see "Groups and Interventions" section for sub-group descriptions).
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Gregory Sawicki, MD, MPH | Boston Children's Hospital | Principal Investigator |
| Kristin Riekert, PhD | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins University | Baltimore | Maryland | 21205 | United States | ||
| Boston Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33735508 | Derived | Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D013375 | Substance Withdrawal Syndrome |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
To determine if treatment arm assignment influenced decision-making about using or not using dornase alfa and/or hypertonic saline post-trial participation |
| from day one to approximately month 8 |
| Change in Participant's Level of Satisfaction in Interviews | To determine pwCF's level of satisfaction with the decision to use or not use dornase alfa and/or hypertonic saline post-trial participation | from day one to approximately month 8 |
| Change in Participant Perceptions (2) |
To determine if individuals who withdrew from the SIMPLIFY study have a different perception about treatment burden and the factors that affect decisions to discontinue treatments. |
| from day one to approximately month 8 |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D019966 | Substance-Related Disorders |
| D064419 | Chemically-Induced Disorders |
| D001523 | Mental Disorders |