Cystic Fibrosis MedDRA version: 19.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Code: VX-371 in hypertonic saline
Pharmaceutical Form: Inhalation solution
INN or Proposed INN: Not yet assigned
Current Sponsor code: VX-371
Sponsors
Vertex Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion 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 FEV1 of =40 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •History of any comorbidity, which in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. •Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject. •An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug). •A 12 lead ECG demonstrating QTcF >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 HS •Known hypersensitivity or history of intolerance to Orkambi. •Pregnant and nursing females. •Subjects who have participated in Parion Sciences Study PS-G201.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline (HS) compared to HS alone in subjects with cystic fibrosis (CF) who are =12 years of age, homozygous for the F508del-CFTR mutation, and being treated with Orkambi;Secondary Objective: To investigate the pharmacokinetics (PK) of VX-371 in subjects with CF who are =12 years of age, homozygous for the F508del-CFTR mutation, and being treated with Orkambi; Timepoint(s) of evaluation of this end point: •Safety from baseline up to 28 days post last administration of study drug, up to 12 Weeks. •Efficacy from study baseline at Day 28 in each Treatment Period ; Primary end point(s): •Results of safety and tolerability assessments of adverse events (AEs), spirometry, clinical laboratory values (urine, serum and plasma chemistry, and hematology), standard 12-lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations •Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •PK parameters for VX-371;Timepoint(s) of evaluation of this end point: •From study baseline at Day 28 in each Treatment Period | — |
Countries
Czech Republic, France, Ireland, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Inc.
Outcome results
None listed