Qualitative Understanding of Experiences With the SIMPLIFY Trial

NCT04320381 | Completed

• 2 other identifiers: IRB-P00034269STRC-113-20-01
• Study Type: observational
• Target: 115
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Cystic Fibrosis; Drug Withdrawal

Keywords

qualitative; treatment withdrawal

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (3)

• Change in Participant's Perceptions in Interviews

  from day one to approximately month 8

• Change in Participant's Decision-making in Interviews

  from day one to approximately month 8

• Change in Participant's Level of Satisfaction in Interviews

  from day one to approximately month 8

Other Outcomes (2)

• Change in Participant Perceptions

  from day one to approximately month 8

• Change in Participant Perceptions (2)

  from day one to approximately month 8

Study Arms (5)

Group A

subjects in the hypertonic saline study randomized to discontinue or maintain therapy

Behavioral: Qualitative Interview

Group B

subjects in the dornase alfa study randomized to discontinue or maintain therapy

Behavioral: Qualitative Interview

Group C

subjects who were randomized but withdrew early from the SIMPLIFY Study.

Behavioral: Qualitative Interview

Group D

subjects in the hypertonic saline study randomized to discontinue or maintain therapy with FEV1% predicted between 40 and \<60%

Behavioral: Qualitative Interview

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)

Behavioral: Qualitative Interview

Interventions

Qualitative Interview BEHAVIORAL

2 Semi-Structured Qualitative Interviews

Group A; Group B; Group C; Group D; Group E

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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).

You may qualify if:

• Patient Participant:
• Participant was randomized into the SIMPLIFY study
• Person with CF is ≥ 14 years of age at the time of randomization into SIMPLIFY
• Is English speaking
• 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
• Participant has provided contact information via the QUEST portal
• Is willing and able to adhere to the study requirements
• Provides consent (or assent if applicable) for the QUEST study
• Completed participation in the SIMPLIFY study (i.e. completed through Visit 3 or early withdrawal, if applicable)
• Caregiver Participant:
• 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
• Is English speaking,
• Is willing and able to adhere to the study requirements
• Provides consent for the QUEST study

You may not qualify if:

• An individual who meets any of the following criteria will be excluded from participation in this study:
• \. Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Johns Hopkins University: collaborator

Study Sites (2)

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• 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.

  PMID: 33735508 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis; Substance Withdrawal Syndrome

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Substance-Related Disorders; Chemically-Induced Disorders; Mental Disorders

Study Officials

• Gregory Sawicki, MD, MPH

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

• Kristin Riekert, PhD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Co-Principal Investigator

Study Record Dates

• First Submitted: March 23, 2020
• First Posted: March 25, 2020
• Study Start: December 23, 2020
• Primary Completion: September 16, 2022
• Study Completion: September 16, 2022
• Last Updated: August 25, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)