Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor (SNO-7)

Nivalis Therapeutics

Status and phase

Unknown

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: Placebo

Drug: Cavosonstat

Study type

Interventional

Funder types

Industry

Identifiers

NCT02724527

N91115-2CF-06

Details and patient eligibility

About

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).

Full description

Assess the effect of Cavosonstat (N91115) on lung function when added to preexisting treatment with ivacaftor in adult patients with CF 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).

Enrollment

19 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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