Phase 2
Completed N=311
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT01225211 ↗Enrolled (actual)
311
Serious AEs
6.5%
Results posted
Oct 2015
Primary outcomePrimary: Cohort 1: Safety and Tolerability Based on Adverse Events (AEs) — 12; 29; 15; 14 participants
Summary
The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cohort 1: Safety and Tolerability Based on Adverse Events (AEs) |
12; 29; 15; 14; 12; 0 | — |
| PRIMARY Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs) |
23; 18; 18; 37; 7; 20 | — |
| PRIMARY Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs |
53; 52; 5; 9 | — |
| PRIMARY Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21 |
-2.131; -9.128; 0.548 | 0.267 |
| PRIMARY Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56 |
0.321; -1.043; -2.900; -1.240; -2.154; 1.627 | 0.680 |
| PRIMARY Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56 |
-1.23; -0.62 | 0.5978 |
| SECONDARY Cohort 1: Absolute Change From Baseline in Sweat Chloride at Day 14 |
-4.442; -1.668 | — |
| SECONDARY Cohort 2 And 3: Absolute Change From Baseline in Sweat Chloride at Day 14 |
-6.490; -5.901; -9.442; -3.137; -9.179; 0.048 | — |
| SECONDARY Cohort 4: Absolute Change From Baseline in Sweat Chloride at Day 56 |
-0.78; -11.82 | — |
| SECONDARY Cohort 1: Absolute Change From Day 14 in FEV1 at Day 21 |
0.128; 0.015; -0.046 | — |
| SECONDARY Cohort 1: Absolute Change From Day 14 in ppFEV1 at Day 21 |
3.46; 0.63; -1.44 | — |
| SECONDARY Cohort 2 and 3: Absolute Change From Day 28 in ppFEV1 at Day 56 |
1.96; 1.99; 6.15; 2.29; 6.09; -1.57 | — |
| SECONDARY Cohort 2 and 3: Relative Change From Day 28 in ppFEV1 at Day 56 |
3.13; 2.98; 9.70; 4.30; 8.24; -2.05 | — |
| SECONDARY Cohort 2 and 3: Absolute Change From Baseline in ppFEV1 at Day 28 and 56 |
0.21; -1.35; -2.62; -3.82; -4.52; -0.03 | — |
| SECONDARY Cohort 2 and 3: Relative Change From Baseline in FEV1 at Day 28 and 56 |
0.24; -1.15; -3.13; -5.46; -6.39; 1.89 | — |
| SECONDARY Cohort 4: Relative Change From Baseline in Percent Predicted FEV1 at Day 56 |
-2.20; -0.69 | — |
| SECONDARY Cohort 2 and 3: Absolute Change From Day 28 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 56 |
3.3; 7.9; 8.9; 5.5; 11.2; -8.6 | — |
| SECONDARY Cohort 4: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 56 |
-0.82; 5.66 | — |
| SECONDARY Cohort 4: Absolute Change From Baseline in Body Mass Index (BMI) at Day 56 |
0.08; -0.04 | — |
| SECONDARY Cohort 4: Absolute Change From Baseline in Weight at Day 56 |
0.16; -0.11 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female participants with confirmed diagnosis of CF
- Must have the F508del-CFTR mutation on at least 1 allele.
- FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age, gender, and height (Hankinson standards (Cohort 4)
- Participant of child-bearing potential and who are sexually active must meet the contraception requirements
Exclusion Criteria
- History of any illness or condition 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 (e.g., cirrhosis with portal hypertension).
- An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first dose of study drug.
- History of solid organ or hematological transplantation.
- History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates.
- Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout
- Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception
- Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3
- Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit
- Heterozygous participants who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4
- Evidence of lens opacity or cataract as determined by the ophthalmologic examination (Cohort 4 only)
Data sourced from ClinicalTrials.gov (NCT01225211). 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.