Phase 2
Completed N=142
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT02709109 ↗Enrolled (actual)
142
Serious AEs
7.9%
Results posted
Jul 2021
Primary outcomePrimary: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs — 65; 66; 32; 28 Participants
Summary
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation, and being treated with Orkambi
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs |
65; 66; 32; 28; 8; 4 | — |
| PRIMARY Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 |
0.1; -0.1; -0.8; 0.8 | 0.8173 |
| SECONDARY Plasma Concentrations of VX-371 |
0.00; 0.18; 2.57; 5.64; 1.63; 2.77 | — |
| SECONDARY Urine Concentrations of VX-371 |
0.0745; 0.00; 7.78; 22.1; 41.0; 69.6 | — |
Eligibility Criteria
Inclusion Criteria
- Willing and able to use the delivery device as directed by the study manual.
- Confirmed diagnosis of CF, defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis.
- Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result is not received before randomization, a previous CFTR genotype lab report may be used to establish eligibility.
- Percent predicted forced expiratory volume at one second (FEV1) of ≥40 to 450 msec at the Screening Visit.
- History of solid organ or hematological transplantation.
- Used diuretics or renin-angiotensin aldosterone system antihypertensive drugs in the 28 days prior to Screening or an anticipated need for any of these medications during the study.
- Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit.
- Inability to withhold short-acting, long-acting, or once-daily, long-acting bronchodilator use for 4, 12, or 24 hours prior to clinic visit, respectively.
- History of significant intolerance to inhaled saline, or intolerance to the single dose of saline at Screening
- Known hypersensitivity or history of intolerance to Orkambi.
- Pregnant and nursing females.
- Subjects who have participated in Parion Sciences Study NCT02343445.
Data sourced from ClinicalTrials.gov (NCT02709109). 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.