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Drug & research development » Clinical trials » NCT01161537

Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation

Official title: A Phase 2, Single-Blind, Placebo-Controlled Study to Evaluate the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis, the G551D Mutation, and FEV1 ≥40% Predicted

Clinical Trials gov number: NCT01161537

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Purpose: A 2-part study to evaluate the effect of ivacaftor on hyperpolarized helium-3 magnetic resonance imaging (3He-MRI), and to evaluate the safety and efficacy of ivacaftor in CF patients aged ≥12 years  who have the G551D-CFTR mutation. Part A is a single-blind, placebo-controlled study that includes 4 weeks of ivacaftor treatment and 4 weeks of placebo treatment. Part B is an open-label, 48 week study of long-term effect of ivacaftor on hyperpolarized 3He-MRI.

Phase: 2

Type: Interventional, non-randomized, single-blind, single group assignment

Study sponsor: Vertex Pharmaceuticals Incorporated; Cystic Fibrosis Foundation Therapeutics

  • Male or female with cystic fibrosis
  • Must have the G551D-CFTR mutation on ≥1 allele
  • FEV1 ≥40% of predicted normal for age, gender, and height at screening
  • Must be able to swallow tablets

Intervention
Ivacaftor

Geographical Location
United States

Number of Participants
≤50 (≥12 years)

Primary Endpoint

Part A:

  • Change from baseline in total ventilation defect defined by hyperpolarized helium 3 magnetic resonance imaging (3He-MRI) at Day 43

Part B:

  • Change from baseline in total ventilation defect defined by 3He-MRI at Week 48

Secondary Endpoint

Part A:

  • Number of participants with adverse events (AEs), serious adverse events (SAEs) and related AEs (Day 1 up to Day 57)
  • Change from baseline (BL) in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43
  • Absolute change from BL in sweat chloride at Day 43
  • Absolute change from BL in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 43

Part B:

  • Number of participants with AEs, SAEs and related AEs (Day 1 up to Week 48)
  • Change from BL in FEV1 at Week 48
  • Absolute change from BL in sweat chloride at Week 48
  • Absolute change from BL in in CFQ-R Respiratory Domain Score At Week 48

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  • Eperimental arm:

Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study.

Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects.

View Trial Results