Trial Outcomes & Findings for PTC Study to Evaluate Ataluren in Combination With Ivacaftor (NCT NCT03256968)

Last Updated: 2020-03-26

Results Overview

effect of ataluren on lung function as assessed by spirometry and measured by percentage of Liters (minimum value would be .0% Liters and maximum value of liters is dependent from person to person). The higher the value the better the outcome. The measure will include the change from baseline to one year to report a change between the two measurement points

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

1 participants

Primary outcome timeframe

1 year

Results posted on

2020-03-26

Participant Flow

Participant milestones

Participant milestones
Measure	Ataluren Administration dose of the drug administered (mg/kg body weight) Ataluren: ataluren
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PTC Study to Evaluate Ataluren in Combination With Ivacaftor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ataluren Administration n=1 Participants dose of the drug administered (mg/kg body weight) Ataluren: 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
Age, Continuous	31 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	Ataluren Administration n=1 Participants dose of the drug administered (mg/kg body weight) Ataluren: ataluren
FEV1 as a Measure of Lung Function	.35 percentage of liters

Adverse Events

Ataluren Administration

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ataluren Administration n=1 participants at risk dose of the drug administered (mg/kg body weight) Ataluren: ataluren
Respiratory, thoracic and mediastinal disorders increased cough	100.0% 1/1 • Number of events 1 • 1 year

Additional Information

Steven Rowe

Universtiy of Alabama at Birmingham

Phone: 205-975-9776

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place