PK Study of N91115 in Cystic Fibrosis Patients

• Status: Completed
• Trial end date: 2015-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Nivalis Therapeutics, Inc.

Criteria

Inclusion Criteria:

1. Confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation

2. Weight ≥ 40 kg at screening

3. FEV1 ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre or post-bronchodilator value, at screening

4. Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening

Exclusion Criteria:

1. 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

2. 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

3. 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

4. History, including the screening assessment, of ventricular tachycardia or ventricular arrhythmias

5. History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec)

6. History of solid organ or hematological transplantation

7. History of alcohol abuse or drug addiction (including cannabis, cocaine, and opioids) in the year prior to screening

8. Use of continuous (24 hr/day) or nocturnal supplemental oxygen