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

NCT ID: NCT02724527

Last Updated: 2016-11-21

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-04-30
• Study Completion Date: 2017-04-30

Brief Summary

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).

Detailed 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).

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; N91115; Cavosonstat

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Placebo

Matching capsule (BID administration Q12H)

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Matched Placebo capsule

Cavosonstat (N91115) 400 mg

Cavosonstat (N91115) at 400 mg dose (100 mg x 4 capsules) (BID administration Q12H)

Group Type: EXPERIMENTAL

Cavosonstat

Intervention Type: DRUG

CFTR modulator that stabilizes CFTR

Interventions

Cavosonstat

CFTR modulator that stabilizes CFTR

Intervention Type: DRUG

Placebo

Matched Placebo capsule

Intervention Type: DRUG

Other Intervention Names

N91115; Control

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nivalis Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

James Chmiel, MD

Role: PRINCIPAL_INVESTIGATOR

Rainbow Babies and Children's Hospital/ University

Locations

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

Countries

United States

Other Identifiers

N91115-2CF-06

Identifier Type: -

Identifier Source: org_study_id