Trial Outcomes & Findings for Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (NCT NCT04378153)

Last Updated: 2024-12-04

Results Overview

The number of participants completing the run-in period of the study and not meeting any of the exclusion criteria during the run-in period.

Recruitment status

COMPLETED

Target enrollment

987 participants

Primary outcome timeframe

2-week run-in period

Results posted on

2024-12-04

Participant milestones
Measure	Master Study Run-in These numbers represent the number of participants enrolled in the 2 week run-in period to be enrolled in a SIMPLIFY sub study. Individual Sub Study Enrollment can be found in: Hypertonic Saline Study Record * NCT06350461 * Brief Title: Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study (SIMPLIFY-HS) Dornase Alfa Study Record * NCT06350474 * Brief Title: Impact of Discontinuing Dornase Alfa in People With CF on Highly Effective CFTR Modulator Therapy-A SIMPLIFY Sub-Study (SIMPLIFY-DN)
Overall Study STARTED	987
Overall Study COMPLETED	903
Overall Study NOT COMPLETED	84

Withdrawal data not reported

Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy

Baseline characteristics by cohort
Measure	Master Study Run-in n=987 Participants Participants enrolled in the run-in phase of the master protocol.
Age, Continuous	22.1 years STANDARD_DEVIATION 10.1 • n=5 Participants
Sex: Female, Male Female	480 Participants n=5 Participants
Sex: Female, Male Male	507 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	67 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	920 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	950 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	12 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaskan Native	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other, More than One Race, or Unknown/Not Reported	22 Participants n=5 Participants
FEV1 (% Predicted)	96.7 Percent Predicted STANDARD_DEVIATION 16.8 • n=5 Participants
Current Medication Use Dornase Alfa Only	232 Participants n=5 Participants
Current Medication Use Hypertonic Saline Only	65 Participants n=5 Participants
Current Medication Use Both Hypertonic Saline and Dornase Alfa	690 Participants n=5 Participants

Timeframe: 2-week run-in period

The number of participants completing the run-in period of the study and not meeting any of the exclusion criteria during the run-in period.

Outcome measures
Measure	Master Study Run-in n=987 Participants Participants enrolled in the run-in phase of the master protocol.
Number of Participants Eligible to be Assessed for Sub-study Enrollment	870 Participants

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Adverse event data not reported

Adverse event data not reported

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