Safety, Tolerability and Efficacy of S-1226 in Cystic Fibrosis and Non CF Bronchiectasis

Summary

Participation Requirements

Description

This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n?12) will be enrolled. The study will consist of a screening period, a run-in and two dosing and evaluation periods (with a minimum two-da...

This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n?12) will be enrolled. The study will consist of a screening period, a run-in and two dosing and evaluation periods (with a minimum two-day break in between) and a follow-up period. The dosing and evaluation period of the study is divided into two consecutive components: Dose escalation study - This segment of the treatment period is designed to assess the safety and tolerability of escalating doses of S-1226 (4%, 8% and 12%) in those with mild-moderate CF and non CF bronchiectasis Daily dosing study - This segment of the treatment period is designed to assess the short term (5 day) safety and tolerability of twice daily administration of a fixed dose of S-1226 in subjects with mild-moderate CF and non CF bronchiectasis. The primary aim of this study of S-1226 is to capture information to inform follow-up S-1226 clinical studies in CF and non CF bronchiectasis. Important in this regard will be data on safety, tolerability, dosing and efficacy. Specifically, regarding efficacy, the investigators want to examine for optimal dose ranging and magnitude of the effect to determine the power calculations for any follow-up studies and to determine which outcome measures best reflect the effects of the drug. Because the study is investigational in nature, it will be un-blinded and information will be analyzed as it is collected. During the screening and the run-in period, the subjects will have safety assessments and all eligibility criteria confirmed. The run-in period involves meeting with the study subjects 1 - 2 weeks prior to the start of the treatment for a physical exam, concomitant medication review, spirometry and information regarding the study. The consent process will also take place at this stage. The dose escalation period will include 3 days of twice daily treatment of the study drug with a washout period in between. The dose of CO2 in S-1226 will be sequentially escalated for the particular subject from the starting dose of 4% CO2. Planned subsequent dose levels are 8% and 12% CO2, although doses may be adjusted within this range based on safety and tolerability data from the completed dosing days. Subjects will not be dosed with a higher CO2 level until drug administration of the preceding dose level has been completed and safety data reviewed by the Safety Committee and a decision taken to proceed or not with the next sequential dose level. There will be a washout period between the dose escalation and the daily dosing study. The daily dosing study will include 5 days of twice daily treatment of the study drug based on the results of the dose escalation study. Lung function tests will be performed before and after the treatment. Subjects in the Home Extension Study will receive S-1226 twice daily for 28 consecutive days. Subjects will monitor daily symptom scores. Oxygen saturation values, as determined by pulse oximetry, will be monitored 2 minutes prior to therapy, during therapy and for up to 30 minutes post therapy. Pulmonary function studies will be assessed weekly by the designated respiratory therapist between 4-7 pm. The follow-up phase will occur 10-14 days after completion of the final dose. This will involve a clinical exam, spirometry and completion of CFQ-R.

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