Qualitative Understanding of Experiences With the SIMPLIFY Trial

Cystic Fibrosis Clinical Trial

— QUEST  

Official title:

Qualitative Understanding of Experiences With the SIMPLIFY Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04320381
• Other study ID #: IRB-P00034269
• Secondary ID: STRC-113-20-01
• Status: Completed
• Start date: December 23, 2020
• Est. completion date: September 16, 2022

Study information

• Verified date: August 2023
• Source: Boston Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 115
• Est. completion date: September 16, 2022
• Est. primary completion date: September 16, 2022
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 14 Years and older

Eligibility

Inclusion Criteria:

Patient Participant:

1. Participant was randomized into the SIMPLIFY study

2. Person with CF is = 14 years of age at the time of randomization into SIMPLIFY

3. Is English speaking

4. Participant has provided consent (or assent if applicable) in the SIMPLIFY study to provide contact information and share data from the SIMPLIFY study with the QUEST study team

5. Participant has provided contact information via the QUEST portal

6. Is willing and able to adhere to the study requirements

7. Provides consent (or assent if applicable) for the QUEST study

8. Completed participation in the SIMPLIFY study (i.e. completed through Visit 3 or early withdrawal, if applicable)

Caregiver Participant:

1. Lives in the same household as a child with CF who is = 14 years of age but <18 years of age who meets the eligibility requirements for the QUEST study

2. Is English speaking,

3. Is willing and able to adhere to the study requirements

4. Provides consent for the QUEST study

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Drug Withdrawal
• Substance Withdrawal Syndrome

Intervention

Behavioral:
• Qualitative Interview
2 Semi-Structured Qualitative Interviews

Locations

Country	Name	City	State
United States	Johns Hopkins University	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Boston Children's Hospital	Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	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
Primary	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)
Primary	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
Secondary	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
Secondary	Change in Participant's Decision-making in Interviews	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
Secondary	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