Trial record 1 of 1 for:
NCT02589236
Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation (SNO-6)
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| ClinicalTrials.gov Identifier: NCT02589236 |
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Recruitment Status :
Completed
First Posted : October 28, 2015
Last Update Posted : January 10, 2017
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Sponsor:
Nivalis Therapeutics, Inc.
Collaborator:
Medidata Solutions
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.
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Brief Summary:
This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: Cavosonstat Drug: Placebo | Phase 2 |
Primary Objective:
- Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR mutation
Secondary Objectives:
- Assess the effect of N91115 added to lumacaftor/ivacaftor on safety
- Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of N91115, lumacaftor, and ivacaftor
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 138 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Care Provider, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor |
| Study Start Date : | November 2015 |
| Actual Primary Completion Date : | December 2016 |
| Actual Study Completion Date : | December 2016 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
| Arm | Intervention/treatment |
|---|---|
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Placebo Comparator: Placebo
Placebo Capsule
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Drug: Placebo
Control sample with only capsule excipients and fillers
Other Name: control |
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Experimental: Cavosonstat (N91115) 200 mg
Cavosonstat (N91115) 200 mg twice daily (BID)
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Drug: Cavosonstat
GSNOR inhibitor
Other Name: N91115 |
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Experimental: Cavosonstat (N91115) 400 mg
Cavosonstat (N91115) 400 mg BID
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Drug: Cavosonstat
GSNOR inhibitor
Other Name: N91115 |
Primary Outcome Measures :
- Absolute change from baseline in percent predicted FEV1 (ppFEV1) [ Time Frame: From baseline to 12 weeks ]Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
Secondary Outcome Measures :
- Relative change from baseline in ppFEV1 [ Time Frame: baseline to 12 weeks ]Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
- Absolute change from baseline in sweat chloride [ Time Frame: baseline to 12 weeks ]A sweat chloride measurement on the skin at study start and after 12 weeks of N91115
- Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale) [ Time Frame: baseline to 16 weeks ]Comparison of the Questionnaire from study start to 16 weeks
- Absolute change from baseline in body mass index (BMI) [ Time Frame: baseline to 12 weeks ]Assessment of change in body mass index from study start to after 12 weeks of N91115
- Absolute change from baseline in Patient Global Impression of Change (PGIC) [ Time Frame: baseline to 12 weeks ]Patient reported outcome journal
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]) [ Time Frame: baseline to 16 weeks ]Any adverse events assessment including clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, or vital sign changes
- Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor [ Time Frame: baseline to 12 weeks ]Maximum Plasma Concentration [Cmax] measurements of N91115, lumacaftor and ivacaftor
- Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor [ Time Frame: baseline to 12 weeks ]AUC measurements of N91115, lumacaftor and ivacaftor
Other Outcome Measures:
- Number of pulmonary exacerbations [ Time Frame: baseline to 12 weeks ]Assessment of number of pulmonary exacerbations at baseline compared through 12 weeks
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior to Day 1 (start of dosing)
- A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)
- Body weight ≥ 40 kg
- ppFEV1 40 - 85 % predicted (inclusive) at screening
- Oxygen saturation ≥ 90% breathing ambient air at screening
Exclusion Criteria:
- Any acute infection that requires treatment or hospitalization within 2 weeks of Study Day 1
- Colonization with organisms associated with more rapid decline in pulmonary status, such as 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
- Are pregnant, planning a pregnancy, or breast-feeding at screening
- Blood hemoglobin < 10 g/dL at screening
- Serum albumin < 2.5 g/dL at screening
- Abnormal liver function defined as ≥ 3 x upper limit of normal (ULN)
- History of abnormal renal function within 3 months of screening
- 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
- History of solid organ or hematological transplantation
- History of alcohol abuse or drug abuse
- Ongoing participation in another therapeutic clinical trial
- Use of continuous (24 hr/day) or nocturnal supplemental oxygen
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02589236
Locations
Show 46 study locations
Sponsors and Collaborators
Nivalis Therapeutics, Inc.
Medidata Solutions
Investigators
| Principal Investigator: | Scott Donaldson, MD | University of North Carolina, Chapel Hill |
| Responsible Party: | Nivalis Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT02589236 |
| Other Study ID Numbers: |
N91115-2CF-05 |
| First Posted: | October 28, 2015 Key Record Dates |
| Last Update Posted: | January 10, 2017 |
| Last Verified: | January 2017 |
Keywords provided by Nivalis Therapeutics, Inc.:
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Cystic Fibrosis N91115 Cavosonstat |
Additional relevant MeSH terms:
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Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |