A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Recruiting in Palo Alto (17 mi)
+47 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Waitlist Available
Sponsor: Vertex Pharmaceuticals Incorporated
No Placebo Group
Prior Safety Data
Breakthrough Therapy
Trial Summary
What is the purpose of this trial?
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
Research Team
Eligibility Criteria
Inclusion Criteria
Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
On ivacaftor therapy
FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
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Treatment Details
Interventions
- VX-561 (Other)
Participant Groups
5Treatment groups
Experimental Treatment
Active Control
Group I: VX-561: 50 mgExperimental Treatment2 Interventions
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
Group II: VX-561: 250 mgExperimental Treatment2 Interventions
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
Group III: VX-561: 25 mgExperimental Treatment2 Interventions
Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
Group IV: VX-561: 150 mgExperimental Treatment2 Interventions
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
Group V: IvacaftorActive Control2 Interventions
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Children's Hospital of Pittsburgh of UPMCPittsburgh, PA
Children's Hospital of PhiladelphiaPhiladelphia, PA
National Jewish HealthDenver, CO
University of Miami Miller School of MedicineMiami, FL
More Trial Locations
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Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
Trials
267
Patients Recruited
36,100+