Phase 1
Completed N=66
Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT01746784 ↗Enrolled (actual)
66
Serious AEs
6.1%
Results posted
Nov 2014
Primary outcomePrimary: Safety and Tolerability — 11; 4; 6; 4 participants
Summary
The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult cystic fibrosis subjects who are homozygous for the F508del-CFTR mutation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability |
11; 4; 6; 4; 12; 6 | — |
| SECONDARY Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) |
1.5; -0.5; -0.2; -0.7; 0.5 | — |
| SECONDARY Change in Biomarkers of CFTR Function |
-0.2; -2.3; -2.9; -0.1; 2.9 | — |
Eligibility Criteria
Inclusion Criteria
- Homozygous for F508del-CFTR gene
- Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
- Body weight ≥ 40 kg
- FEV1 ≥ 40% predicted
- Oxygen saturation ≥ 90% breathing ambient air
- Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments
- Negative pregnancy test for women of child bearing potential
- Sexually active subjects of child bearing potential willing to follow contraception requirements
Exclusion Criteria
- Previous enrollment in another cohort for this study.
- Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment within 4 weeks of Study Day 1.
- Any change in chronic therapies for CF lung disease within 4 weeks of Study Day 1.
- Blood hemoglobin 450 msec).
- History of solid organ or hematological transplantation.
- Intranasal medication changes within 14 days prior to Study Day 1
- Required Use of continuous (24 hr/d) or nocturnal supplemental oxygen.
- Concomitant use of any inhibitors or inducers of CYP3A4.
Data sourced from ClinicalTrials.gov (NCT01746784). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.