Phase 2 Completed N=40 Randomized Triple-blind Treatment

Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

Source: ClinicalTrials.gov NCT02070744 ↗

Enrolled (actual)

Serious AEs

27.3%

Results posted

Apr 2018

Primary outcomePrimary: PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 6; 5; 15; 13 Participants

Summary

The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation

Outcome Measures

Outcome	Result	p-value
PRIMARY PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	6; 5; 15; 13; 1; 2	—
PRIMARY OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs	25; 6	—
SECONDARY PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12	0.9; -0.1; 3.0; 1.9	—
SECONDARY OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40	2.7	—
SECONDARY PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12	2.6; 1.0; 6.0; 4.2	—
SECONDARY OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40	6.1	—
SECONDARY PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12	-10.6; 2.9; -4.7; 0.8	—
SECONDARY OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40	-6.6	—
SECONDARY PC Phase: Absolute Change From Baseline in Body Weight at Week 12	1.0; 1.6; 0.5; 0.3	—
SECONDARY OLE Phase: Absolute Change From Baseline in Body Weight at Week 40	1.0	—
SECONDARY PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12	0.38; 0.54; 0.18; 0.11	—
SECONDARY OLE Phase: Absolute Change From Baseline BMI at Week 40	0.33	—
SECONDARY PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12	5.6; -4.6; 1.0; 0.4	—
SECONDARY OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40	-0.6	—
SECONDARY PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA	4890; 6460; 1490; 1210	—
SECONDARY PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661	84900; 75500	—
SECONDARY PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA	14700; 10100	—
SECONDARY PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA	2.48; 3.23; 3.59; 4.00	—

Eligibility Criteria

Inclusion Criteria

Homozygous for the F508del CFTR mutation
FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
Stable CF disease as judged by the investigator

Exclusion Criteria

History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant
Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02070744). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.