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Purpose

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Exclusion Criteria

  • History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Ivacaftor 		Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
  • Drug: IVA
    150-mg film-coated tablet for oral administration.
    Other names:
    • VX-770
    • Ivacaftor
  • Drug: Placebo
    Placebos matched to VX-561.
Experimental
VX-561: 25 mg 		Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
  • Drug: VX-561
    VX-561 tablets for oral administration.
    Other names:
    • CTP-656
    • Deutivacaftor (D-IVA)
  • Drug: Placebo
    Placebo matched to IVA.
Experimental
VX-561: 50 mg 		Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
  • Drug: VX-561
    VX-561 tablets for oral administration.
    Other names:
    • CTP-656
    • Deutivacaftor (D-IVA)
  • Drug: Placebo
    Placebo matched to IVA.
Experimental
VX-561: 150 mg 		Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
  • Drug: VX-561
    VX-561 tablets for oral administration.
    Other names:
    • CTP-656
    • Deutivacaftor (D-IVA)
  • Drug: Placebo
    Placebo matched to IVA.
Experimental
VX-561: 250 mg 		Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
  • Drug: VX-561
    VX-561 tablets for oral administration.
    Other names:
    • CTP-656
    • Deutivacaftor (D-IVA)
  • Drug: Placebo
    Placebo matched to IVA.

More Details

Status
Completed
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.