Cystic FibrosisQUESTNCT04320381
Qualitative Understanding of Experiences With the SIMPLIFY Trial
An observational study of Cystic Fibrosis and Drug Withdrawal, run by Boston Children's Hospital at 2 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 14 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Drug Withdrawal
- What is being tested?
- Qualitative Interview
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 115 participants
- Where
- 2 sitesUnited States
- Study dates
- 23 Dec 2020to 16 Sep 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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).
Who may be able to join
- 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
Show all 14Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Johns Hopkins University
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
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.
Read the detailed 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.
- Conditions
- Cystic Fibrosis, Drug Withdrawal
- Being tested
- Qualitative Interview
- Keywords
- qualitative, treatment withdrawal
- Medical subject headings
- Cystic Fibrosis, Substance Withdrawal Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Qualitative InterviewBehavioural
2 Semi-Structured Qualitative Interviews
| Study group | Role | What they receive |
|---|---|---|
| Group A | subjects in the hypertonic saline study randomized to discontinue or maintain therapy Receives: Behavioral: Qualitative Interview | |
| Group B | subjects in the dornase alfa study randomized to discontinue or maintain therapy Receives: Behavioral: Qualitative Interview | |
| Group C | subjects who were randomized but withdrew early from the SIMPLIFY Study. Receives: Behavioral: Qualitative Interview | |
| Group D | subjects in the hypertonic saline study randomized to discontinue or maintain therapy with FEV1% predicted between 40 and <60% Receives: 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) Receives: Behavioral: Qualitative Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: to be conducted 4 months following the date of the first interview
Secondary outcomes (3)
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).
Measured: from day one to approximately month 8
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
Measured: 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
Measured: from day one to approximately month 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2020
- Study start23 Dec 2020
- Primary completion16 Sep 2022
- Study completion16 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 115 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University
- Responsible party
- Principal investigator, Gregory Sawicki, Boston Children's Hospital
- Investigators
- Gregory Sawicki, MD, MPH, Principal investigator, Boston Children's HospitalKristin Riekert, PhD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04320381
- Sponsor's protocol ID
- IRB-P00034269
- Other IDs
- STRC-113-20-01
- Acronym
- QUEST
- Status last verified
- Aug 2023
- First submitted
- 23 Mar 2020
- Last updated
- 25 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 33735508
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Qualitative Understanding of Experiences With the SIMPLIFY Trial
- ClinicalTrials.gov ID
- NCT04320381
- Acronym / sponsor ID
- QUEST / IRB-P00034269
- Conditions
- Cystic Fibrosis, Drug Withdrawal
- Keywords
- qualitative, treatment withdrawal
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- 23 Dec 2020 (actual)
- Primary completion
- 16 Sep 2022 (actual)
- Study completion
- 16 Sep 2022 (actual)
- First posted
- 25 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Group A | – | Behavioral: Qualitative Interviewsubjects in the hypertonic saline study randomized to discontinue or maintain therapy |
| Group B | – | Behavioral: Qualitative Interviewsubjects in the dornase alfa study randomized to discontinue or maintain therapy |
| Group C | – | Behavioral: Qualitative Interviewsubjects who were randomized but withdrew early from the SIMPLIFY Study. |
| Group D | – | Behavioral: Qualitative Interviewsubjects in the hypertonic saline study randomized to discontinue or maintain therapy with FEV1% predicted between 40 and <60% |
| Group E | – | Behavioral: Qualitative Interviewcaregiver participants (parents and legal guardians of eligible patient participants less than 18 years of age who were randomized in the SIMPLIFY study) |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Qualitative Interview2 Semi-Structured Qualitative Interviews | Behavioural | Group A, Group B, Group C, Group D, Group E |
Primary outcome
Interview #1
Time frame: to be conducted at the time of enrollment (post SIMPLIFY study)
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.
Interview #2
Time frame: to be conducted 4 months following the date of the first interview
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.
3 secondary outcomes
Change in Participant's Perceptions in Interviews
Time frame: from day one to approximately month 8
Change in Participant's Decision-making in Interviews
Time frame: from day one to approximately month 8
Change in Participant's Level of Satisfaction in Interviews
Time frame: from day one to approximately month 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2023.
- Derived from this study: PubMed 33735508Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gregory Sawicki, MD, MPH principal investigator
Boston Children's Hospital
Kristin Riekert, PhD principal investigator
Johns Hopkins University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis and Drug Withdrawal, run by Boston Children's Hospital at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Interview #1 (time frame: to be conducted at the time of enrollment (post SIMPLIFY study)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04320381, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2023.