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Drug & research development » Clinical trials » NCT01614470

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation (KONNECTION)

Official title: A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation

Clinical Trials gov number: NCT01614470

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Purpose: To evaluate the efficacy and safety of ivacaftor in CF patients who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).

Phase: 3

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

Study sponsor: Vertex Pharmaceuticals Incorporated; Cystic Fibrosis Foundation Therapeutics

  • Male or female with confirmed diagnosis of CF
  • ≥1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
  • Percent predicted forced expiratory volume in 1 second (FEV1) ≥40% predicted normal for age, sex, and height
  • Minimum weight of 15kg at screening
  • Females of childbearing potential must not be pregnant
  • Willing to comply with contraception requirements

Intervention
Ivacaftor

Geographical Location
Belgium, France, United States

Number of Participants
≤50 (≥6 years)

Primary Endpoint

Part 1:

Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) through Week 8

Part 2:

Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) through 24 Weeks of treatment (Week 36 visit)

Secondary Endpoint

Part 1:

  • Change from baseline (BL) in body mass index (BMI) at Week 8
  • Change from BL in sweat chloride through Week 8
  • Change from BL in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score through Week 8
  • Number of participants with adverse events (AEs) and serious adverse events (SAEs) from signing informed consent up to Week 20

Part 2:

  • Change from BL in BMI at 24 Weeks (Week 36 Visit)
  • Change from BL in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit)
  • Change from BL in CFQ-R Respiratory Domain Score Through 24 Weeks  (Week 36 Visit)
  • Number of participants with AEs and SAEs from Week 20 up to Week 40

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  • Experimental part 1: Ivacaftor First, Then Placebo

Ivacaftor 150 milligram (mg) tablet orally twice daily (q12h) for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily (q12h) for 8 weeks in treatment period 2

 Washout out period of 4 to 8 weeks was maintained between each treatment period.

  • Experimental part 1: Placebo First, Then Ivacaftor

Placebo matched to ivacaftor tablet orally twice daily (q12h) for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily (q12h) for 8 weeks in treatment period 2.

 Washout out period of 4 to 8 weeks was maintained between each treatment period.

  • Experimental part 2: Ivacaftor

Ivacaftor 150 mg tablet orally twice daily (q12h) for 16 weeks.

View Trial Results