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Drug & research development » Clinical trials » NCT01746784

Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (SNO-1)

Official title: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of N6022 to Evaluate Safety and Pharmacokinetics in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (SNO1)

Clinical Trials gov number: NCT01746784

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Purpose: To investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult CF patients who are homozygous for the F508del-CFTR mutation.

Phase: 1

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

Study sponsor: Nivalis Therapeutics, Inc.

  • Homozygous for F508del-CFTR gene
  • Sweat chloride ≥60 mEq/L by quantitative pilocarpine iontophoresis
  • Body weight ≥40 kg
  • FEV1 ≥40% predicted
  • Oxygen saturation ≥90% breathing ambient air
  • Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments
  • Negative pregnancy test for women of child bearing potential
  • Sexually active subjects of child bearing potential willing to follow contraception requirements

Intervention
N6022

Geographical Location
United States

Number of Participants
51-100 (≥18 years)

Primary Endpoint

Safety and tolerability over 7 treatment days and 7 days of follow-up

Secondary Endpoint

From baseline at Day 7

  • Change in percent predicted forced expiratory volume in 1 second (FEV1)
  • Change in biomarkers of CFTR function

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  • Experimental: N6022

Subjects randomized to study drug will receive N6022 by intravenous infusion once per day for 7 days

  • Placebo Comparator: Normal saline

Subjects randomized to placebo will receive normal saline administered intravenously using the same volume as the active drug group

   

View Trial Results