Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations (NCT NCT02971839)

NCT ID: NCT02971839

Last Updated: 2020-08-26

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

From baseline at Day 28

Results posted on

2020-08-26

Participant Flow

Participant milestones

Participant milestones
Measure	VX-561 20 mg Participants received VX-561 20 mg orally once daily for 28 days.	VX-561 100 mg Participants received VX-561 100 mg orally once daily for 28 days.	VX-561 150 mg Participants received VX-561 150 mg orally once daily for 28 days.	Placebo Participants received placebo matched to VX-561 orally once daily for 28 days.	Ivacaftor Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.
Overall Study STARTED	2	2	2	2	3
Overall Study COMPLETED	2	2	2	2	3
Overall Study NOT COMPLETED	0	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	VX-561 20 mg n=2 Participants Participants received VX-561 20 mg orally once daily for 28 days.	VX-561 100 mg n=2 Participants Participants received VX-561 100 mg orally once daily for 28 days.	VX-561 150 mg n=2 Participants Participants received VX-561 150 mg orally once daily for 28 days.	Placebo n=2 Participants Participants received placebo matched to VX-561 orally once daily for 28 days.	Ivacaftor n=3 Participants Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.	Total n=11 Participants Total of all reporting groups
Age, Continuous	24.0 years STANDARD_DEVIATION 1.41 • n=5 Participants	20.0 years STANDARD_DEVIATION 1.41 • n=7 Participants	19.5 years STANDARD_DEVIATION 2.12 • n=5 Participants	29.0 years STANDARD_DEVIATION 2.83 • n=4 Participants	19.7 years STANDARD_DEVIATION 0.58 • n=21 Participants	22.2 years STANDARD_DEVIATION 4.00 • n=10 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants	2 Participants n=21 Participants	9 Participants n=10 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	2 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	1 Participants n=4 Participants	3 Participants n=21 Participants	10 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants	3 Participants n=21 Participants	11 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Sweat Chloride	59.25 millimole per liter (mmol/L) STANDARD_DEVIATION 16.617 • n=5 Participants	82.25 millimole per liter (mmol/L) STANDARD_DEVIATION 27.931 • n=7 Participants	61.50 millimole per liter (mmol/L) STANDARD_DEVIATION 32.527 • n=5 Participants	36.50 millimole per liter (mmol/L) STANDARD_DEVIATION 29.698 • n=4 Participants	71.25 millimole per liter (mmol/L) STANDARD_DEVIATION 37.830 • n=21 Participants	62.15 millimole per liter (mmol/L) STANDARD_DEVIATION 27.355 • n=10 Participants

PRIMARY outcome

Timeframe: From baseline at Day 28

Population: As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, model-based analyses and summary statistics plan were not performed. Therefore, no efficacy summary is provided for this outcome measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline at Day 28

Population: As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, all model-based analyses and summary statistics are no longer applicable and are therefore removed from analysis plan. Therefore no efficacy summary is available for this outcome measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline at Day 28