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Drug & research development » Clinical trials » NCT01531673

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

Official title: A Phase 2, Multicenter, Double-Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Clinical Trials gov number: NCT01531673

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Purpose: To evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation.

Phase: 2

Type: Interventional, randomized, double-blind, parallel assignment

Study sponsor: Vertex Pharmaceuticals Incorporated

  • Male or female with confirmed diagnosis of CF
  • Must have the F508del-CFTR gene mutation in both alleles (Groups 1-6)
  • Group 7 subjects must have the F508del-CFTR mutation on 1 allele, and gating mutation G551D on the second allele and have been on their physician prescribed 150mg ivacaftor q12h for at least 28 days at the screening visit
  • Forced expiratory volume in 1 second (FEV1) 40–90% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at screening
  • Weight >40kg and BMI >18.5
  • Subjects of child-bearing potential and who are sexually active must meet the contraception requirements.

Intervention
Ivacaftor, VX-661

Geographical Location
Canada, Germany, United Kingdom, United States

Number of Participants
101-1000 (≥12 years)

Primary Endpoint

  • Safety and tolerability of active drug vs placebo through Day 56 (measured by incidence of treatment-emergent adverse events, and by clinical laboratory values)
  • Absolute change in sweat chloride from baseline to Day 28

Secondary Endpoint

From baseline to Day 28

  • Change in sweat chloride
  • Change in percent predicted forced expiratory volume in 1 second
  • Change in forced expiratory volume in 1 second
  • Change in Cystic Fibrosis Questionnaire-Revised respiratory domain score

Through 56 days

  • PK parameters of VX-661, ivacaftor, and their respective metabolites in plasma when VX-661 is administered alone and when the 2 drugs are administered together

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  • Experimental group 1: F508d homozygous subjects to receive 10 mg VX-661 monotherapy once daily for 28 days
  • Experimental group 2:
    a) F508d homozygous subjects to receive 30 mg VX-661 monotherapy once daily for 28 days
    b) F508d homozygous subjects to receive 10 mg VX-661 once daily and 150 mg ivacaftor twice daily for 28 days
  • Experimental group 3:
    a) F508d homozygous subjects to receive 100 mg VX-661 monotherapy once daily for 28 days
    b) F508d homozygous subjects to receive 30 mg VX-661 once daily and ivacaftor 150mg twice daily for 28 days
  • Experimental group 4: F508d homozygous subjects to receive 100 mg VX-661 once daily and ivacaftor 150mg twice daily for 28 days
  • Experimental group 5:
    a) F508d homozygous subjects to receive 150 mg VX-661 monotherapy once daily for 28 days.
    b) F508d homozygous subjects to receive 150 mg VX-661 once daily and ivacaftor 150 mg twice daily for 28 days.
  • Experimental group 6:
    a) F508d homozygous subjects to receive 100 mg VX-661 once daily and ivacaftor 50 mg twice daily for 28 days.
    d) F508d homozygous subjects to receive 50 mg VX-661 twice daily and ivacaftor 150 mg twice daily for 28 days.
  • Experimental group 7: F508d/G551D subjects to receive 100 mg VX-661 once daily for 28 days
  • Placebo group 1: F508d homozygous subjects to receive VX-661-placebo monotherapy once daily for 28 days.
  • Placebo group 2:
    a) F508d homozygous subjects to receive VX-661-placebo monotherapy once daily for 28 days
    b) F508d homozygous subjects to receive VX-661-placebo once daily and ivacaftor-placebo twice daily for 28 days.
  • Placebo group 3:
    a) F508d homozygous subjects to receive VX-661-placebo monotherapy once daily for 28 days.
    b) F508d homozygous subjects to receive VX-661-placebo once daily and ivacaftor-placebo twice daily for 28 days.
  • Placebo group 4:F508d homozygous subjects to receive VX-661-placebo once daily and ivacaftor-placebo twice daily for 28 days.
  • Placebo group 5:
    a) F508d homozygous subjects to receive VX-661-placebo monotherapy once daily for 28 days.
    b) F508d homozygous subjects to receive VX-661-placebo once daily and ivacaftor-placebo twice daily for 28 days.
  • Placebo group 6:
    a) F508d homozygous subjects to receive VX-661-placebo once daily and ivacaftor-placebo twice daily for 28 days.
    d) F508d homozygous subjects to receive VX-661-placebo twice daily and ivacaftor-placebo twice daily for 28 days.
  • Placebo group 7: F508d/G551D subjects to receive VX-661-placebo once daily for 28 days
View Trial Results