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Drug & research development » Clinical trials » NCT02275936

Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation (SNO4)

Official title: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Parallel, Group Study of N91115 to Evaluate Safety and Pharmacokinetics in Patients With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation

Clinical Trials gov number: NCT02275936

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Purpose: A Phase 1b study in F508del-CFTR homozygous CF patients to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses near the expected therapeutic exposure level in preparation for Phase 2 studies of N91115 added to the CFTR modulator combination VX-809/ivacaftor when launched.

Phase: 1

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

Study sponsor: Nivalis Therapeutics, Inc.

  • Male or female with confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation based on historical results generated by Ambry Genetics within the past two years or if unavailable, confirmed by testing done within the past 28 days
  • Sweat chloride ≥ 60 (milliequivalents) mEq/L, by quantitative pilocarpine iontophoresis test (QPIT) at screening
  • Weight ≥ 40 kg at screening
  • Forced expiratory volume (FEV1) ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre- or post-bronchodilator value, at screening
  • Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening
  • Hematology, clinical chemistry and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening


Geographical Location
United States

Number of Participants
≤50 (18-80 years)

Primary Endpoint

Safety assessments based on clinical evaluations, laboratory assessments and adverse events (28 days)

Secondary Endpoint

Pharmacokinetic parameters of N91115 and its glucuronide metabolite in plasma (28 days)

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  • Experimental: Group 1 – 50 mg every 12 hour oral dosing of N91115 for 28 days
  • Experimental: Group 2 – 100 mg every 12 hour oral dosing of N91115 for 28 days
  • Experimental: Group 3 – 200 mg every 12 hour oral dosing of N91115 for 28 days
  • Placebo Comparator: Group 4 – Placebo every 12 hour oral dosing of placebo comparator for 28 days
View Trial Results