Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor

Status unconfirmed · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

Cavosonstat (N91115) is being studied as a potential novel therapy for cystic fibrosis (CF), and this study assesses a target population of patients who are heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R).

Key facts

Study ID

NCT02724527

Run by

Nivalis Therapeutics, Inc.

People needed

19

Starts

2016-04-01

Expected to finish

2017-04-01

Last updated by the study team

2016-11-21

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of CF, heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R)
• Have been treated with chronic ivacaftor twice daily for at least 6 months prior to Screening (date of consent) and are currently being treated with commercially available Ivacaftor
• Negative serum pregnancy test
• Weight ≥ 40 kg at screening
• Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening

You may not qualify if…

• Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment that has completed within 2 weeks of Study Day 1 or hospitalization discharge within 2 weeks of Study Day 1
• Recent infection (per investigator discretion) with organisms associated with more rapid decline in pulmonary status, for example: Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus
• Any change in the regimen for chronic therapies for CF lung disease (e.g., Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
• Blood hemoglobin < 10 g/dL at screening
• Serum albumin < 2.5 g/dL at screening
• Abnormal liver or renal function
• History of ventricular tachycardia or other clinically significant ventricular arrhythmias
• History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec for men; > 470 msec for women)
• History of solid organ or hematological transplantation
• History of alcohol abuse or drug abuse (including cannabis, cocaine, and opioids) in the year prior to screening
• Use of continuous (24 hr/day) or nocturnal supplemental oxygen

Where it is running

• National Jewish Health — Denver, Colorado, United States
• Johns Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Columbia University — New York, New York, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Rainbow Babies and Children's Hospital - Case Medical Center — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health and Science University — Portland, Oregon, United States
• Children's Hospital Pittsburgh — Pittsburgh, Pennsylvania, United States
• University of Utah — Salt Lake City, Utah, United States
• Medical Center of Wisconsin — Madison, Wisconsin, United States

Full record on ClinicalTrials.gov

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