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PK Study of N91115 in Cystic Fibrosis Patients (SNO3)

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

N91115

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring CF, F508Del, Cavosonstat, N91115

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation
Weight ≥ 40 kg at screening
FEV1 ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre or post-bronchodilator value, at screening
Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening

Exclusion Criteria:

History of any illness or condition that in the opinion of the investigator could confound the results of the study or pose additional risk when administered IMP
Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment or hospitalizations within 4 weeks of Study Day 1
Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
History, including the screening assessment, of ventricular tachycardia or ventricular arrhythmias
History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec)
History of solid organ or hematological transplantation
History of alcohol abuse or drug addiction (including cannabis, cocaine, and opioids) in the year prior to screening
Use of continuous (24 hr/day) or nocturnal supplemental oxygen

Sites / Locations

University of Alabama @ Birmingham
Children's CO
National Jewish Health

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

N91115

Arm Description

Every 12 hour oral dosing of 50 mg N91115 for 14 days

Outcomes

Primary Outcome Measures

Pharmacokinetics

To assess the pharmacokinetics of N91115 after twice daily (q 12 h) doses given for 14 days by measuring plasma and urine concentrations of N91115 and its primary metabolite.

Secondary Outcome Measures

Safety of dosing twice per day over 14 days

To assess the safety of 14 twice daily (q 12 h) doses of N91115 in F508del-CFTR homozygous CF patients by monitoring, 12 lead ECG, Adverse events, Clinical safety laboratory tests (hematology, clinical chemistry, and urinalysis), Concomitant medications, Abbreviated physical examinations, Hemoglobin oxygen saturation (O2 Sat) by pulse oximetry, Spirometry, Telemetry, Vital signs, Weight

Full Information

NCT ID

NCT02227888

First Posted

August 26, 2014

Last Updated

November 3, 2016

Sponsor

Nivalis Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02227888

Brief Title

PK Study of N91115 in Cystic Fibrosis Patients

Acronym

SNO3

Official Title

A Phase 1, Open-Label Study Evaluating the Pharmacokinetics of N91115 in Cystic Fibrosis Patients

Study Type

Interventional

2. Study Status

Record Verification Date

November 2016

Overall Recruitment Status

Completed

Study Start Date

October 2014 (undefined)

Primary Completion Date

January 2015 (Actual)

Study Completion Date

January 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Nivalis Therapeutics, Inc.

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

This Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials.

Detailed Description

This is an open-label pharmacokinetic (PK) study of twice daily doses of 50 mg of N91115 administered for 14 days in F508del-CFTR homozygous CF patients. Six patients are planned for enrollment. Each patient will undergo screening (Day 28 to Day 3) and, if eligible, will return to the clinical site on Day 1 or optionally on Day 1. Eligibility will be reconfirmed and the patient will be admitted to a clinical research unit (CRU). Patients will stay overnight from Day 1 to Day 2 and will be discharged on Day 2 after all procedures and assessments are completed. Patients will return to the CRU as outpatients on Days 4, 7, 11, 14, and 15 for PK sample collection and other assessments. They will receive an oral dose of investigational medicinal product (IMP), N91115, twice daily on Days 1 through 14 and will be followed for PK through Day 15. Telephone calls to assess safety and ensure compliance with dosing will be made on days that patients do not make clinic visits (Days 3, 5, 6, 8, 9, 10, 12, and 13). Follow-up safety calls will then be made on Day 21 (adverse events) and Day 28 (serious adverse events only). Participation of an individual patient may last approximately 56 days from the time of screening until the end-of-study (Day 28) follow-up call.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

Keywords

CF, F508Del, Cavosonstat, N91115

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

N91115

Arm Type

Experimental

Arm Description

Every 12 hour oral dosing of 50 mg N91115 for 14 days

Intervention Type

Drug

Intervention Name(s)

N91115

Other Intervention Name(s)

Cavosonstat

Intervention Description

S nitrosoglutathione reductase inhibitor

Primary Outcome Measure Information:

Title

Pharmacokinetics

Description

To assess the pharmacokinetics of N91115 after twice daily (q 12 h) doses given for 14 days by measuring plasma and urine concentrations of N91115 and its primary metabolite.

Time Frame

14 Days

Secondary Outcome Measure Information:

Title

Safety of dosing twice per day over 14 days

Description

Time Frame

14 Days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation Weight ≥ 40 kg at screening FEV1 ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre or post-bronchodilator value, at screening Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening Exclusion Criteria: History of any illness or condition that in the opinion of the investigator could confound the results of the study or pose additional risk when administered IMP Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment or hospitalizations within 4 weeks of Study Day 1 Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1 History, including the screening assessment, of ventricular tachycardia or ventricular arrhythmias History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec) History of solid organ or hematological transplantation History of alcohol abuse or drug addiction (including cannabis, cocaine, and opioids) in the year prior to screening Use of continuous (24 hr/day) or nocturnal supplemental oxygen

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Steven Shoemaker, MD

Organizational Affiliation

Nivalis Therapeutics, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

University of Alabama @ Birmingham

City

Birmingham

State/Province

Alabama

ZIP/Postal Code

35294

Country

United States

Facility Name

Children's CO

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

National Jewish Health

City

Denver

State/Province

Colorado

ZIP/Postal Code

80206

Country

United States

12. IPD Sharing Statement

Learn more about this trial

PK Study of N91115 in Cystic Fibrosis Patients

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