Trial Outcomes & Findings for Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations (NCT NCT03256799)

Results Overview

change in lung function as measured by spirometry

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

1 participants

Primary outcome timeframe

Baseline through 48 weeks

Results posted on

2019-07-19

N of 1 trial

there was no multiple arms in this study and no multiple groups

Participant milestones
Measure	Ivacaftor/Ataluren the combination treatment of Ivacaftor/Ataluren were to be given over a 48 week period
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Withdrawal data not reported

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

Baseline characteristics by cohort
Measure	Ivacaftor/Ataluren n=1 Participants Ivacaftor/Ataluren
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Race/Ethnicity, Customized caucasian, non-hispanic	1 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

Timeframe: Baseline through 48 weeks

change in lung function as measured by spirometry

Outcome measures
Measure	Ivacaftor/Ataluren n=1 Participants Ivacaftor/Ataluren combination therapy
Lung Function	35 Liters

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Adverse event data not reported

Other adverse events
Measure	Ivacaftor/Ataluren n=1 participants at risk Ivacaftor/Ataluren
Respiratory, thoracic and mediastinal disorders pulmonary exacerbation	100.0% 1/1 • Number of events 2 • 4 months from March 2017 through July 2017 two pulmonary exacerbations during the N-of-1 trial, both of which occurred following cessation of combination therapy

UAB

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