Trial Outcomes & Findings for Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients (NCT NCT01784419)

Last Updated: 2020-09-29

Results Overview

Sweat chloride concentration measured by pilocarpine iontophoresis, a standard clinical laboratory technique. Sweat collection accomplished with the Wescor Macroduct system. Sweat chloride is measured at the start and end of each study period. There are two study periods during which subjects take either ivacaftor or placebo.

Recruitment status

COMPLETED

Study phase

Target enrollment

10 participants

Primary outcome timeframe

14 +/- 2 days

Results posted on

2020-09-29

Participant Flow

72 patients were screened for eligibility.

All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.

Participant milestones

Participant milestones
Measure	All Study Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Overall Study STARTED	10
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	All Study Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Overall Study Adverse Event	3

Baseline Characteristics

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Study Participants n=7 Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race/Ethnicity, Customized White	5 Participants n=5 Participants
Race/Ethnicity, Customized Persian	1 Participants n=5 Participants
Race/Ethnicity, Customized Latino	1 Participants n=5 Participants
Pancreatic sufficient Yes	5 Participants n=5 Participants
Pancreatic sufficient No	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 14 +/- 2 days

Outcome measures

Outcome measures
Measure	All Study Participants n=7 Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Sweat Chloride Concentration	NA mmol/L Standard Deviation NA The PI has left the institution and the only access to the data is from the publication. The access to this specific data cannot be confirmed and is not available to be reported.

SECONDARY outcome

Timeframe: 14 +/- 2 days

Standard spirometry will be performed at the start and end of each 2 week study period. Subjects will take study drug (ivacaftor or placebo) during each study period.

Outcome measures

Outcome measures
Measure	All Study Participants n=7 Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Spirometry	NA Participants The PI has left the institution and the only access to the data is from publication. The access to this specific data cannot be confirmed and is not available to be reported.

SECONDARY outcome

Timeframe: 14 +/- 2 days

Subjects will perform multibreath washout testing using standard techniques to measure functional residual capacity and lung clearance index at the beginning and end of each study period.

Outcome measures

Outcome measures
Measure	All Study Participants n=7 Participants The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Multibreath Washout Testing	NA Participants The PI has left the institution and the only access to the data is from publication. The access to this specific data cannot be confirmed and is not available to be reported.

Adverse Events

All Study Participants

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	All Study Participants n=10 participants at risk The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course. The placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Respiratory, thoracic and mediastinal disorders Epistaxis	10.0% 1/10 All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.
Eye disorders Injected conjunctiva	10.0% 1/10 All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.
Gastrointestinal disorders Ileus	10.0% 1/10 All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.
Respiratory, thoracic and mediastinal disorders Pulmonary exacerbations	20.0% 2/10 All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.
Infections and infestations Viral infection	10.0% 1/10 All study participants are combined and reported here in one group because the data are reported this way in the only accessible data (in the publication). The PI has left the institution and the sequence specific data cannot be confirmed and are not available to be reported.

Additional Information

Dennis W. Nielson, MD, PhD

University of California, San Francisco

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place