A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis

A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF

The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: PUR118

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • Northern Ireland
    • Belfast, Northern Ireland, United Kingdom, BT9 6AD
      • Celerion

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Males or non-pregnant, non-lactating healthy female.
• Age 18 to 55 years of age (inclusive) on day of signing informed consent.
• Subject has a BMI of 17 or greater.
• Subject has a confirmed diagnosis of cystic fibrosis documented by a compatible clinical or radiographic presentation, and definitive accepted lab and clinical criteria (e.g., sweat chloride > 60 mEq/L or 2 disease causing CFTR mutations) OR is regularly attending a Consultant led clinic where the diagnosis of Cystic Fibrosis is not in doubt.
• Subject has an FEV1 ≥ 45% of predicted at screening.
• Subject has an oxygen saturation >92% on room air as determined by pulse oximetry at screening.
• Subject is a non-smoker or ex-smoker has not smoked for at least six months prior to screening.
• Subject is otherwise medically stable without clinically significant abnormal screening results that depart from their usual baseline level of health as a subject with CF.
• Must be willing and able to communicate in English and participate in the whole study.
• Must provide written informed consent.

Exclusion Criteria:

• Subject has unstable lung disease, as defined by a change in medical regimen during the preceding 2-weeks, or a significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse), or in the opinion of the Investigator, has had a decline in pulmonary status within the last year not considered a part of the usual, chronic progression of CF lung disease and part of their baseline health condition as a subject with CF.
• Subject has had an exacerbation of respiratory symptoms within the past 30 days that required initiation of a new or altered respiratory therapy.
• Subject had either an upper or lower respiratory illness within the 30 days prior to dosing days, or has symptoms from such an illness that have not resolved.
• Subject has a history of lung transplantation.
• Females of child bearing potential who are pregnant, or lactating. Females who are sexually active and either not surgically sterile or not willing to use an acceptable form of contraception
• Clinically significant abnormal biochemistry, haematology or urinalysis not consistent with CF as judged by the Investigator
• Failure to satisfy the Investigator of fitness to participate for any other reason.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: PUR118; low dose and high dose for 3 doses, BID
Experimental: PUR118	Drug: PUR118; low dose and high dose for 3 doses, BID

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and Tolerability	Changes in clinical signs and symptoms from physical examination and chest examination, changes in clinical safety laboratory tests, vital signs, spirometry, lung clearance index, 24-hour sputum weight and volume, oxygen saturation, ECG, telemetry, and adverse events.	4-6 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacodynamics	To assess impact of PUR118 on mobilization and expectorated volume of airway mucus secretions. To evaluate impact of PUR118 on measurements of lung function, including ventilation inhomogeneity, by both spirometry and measurement of the lung clearance index (LCI). To evaluate an impact of PUR118 on sputum, serum and plasma biomarkers related to airway inflammation.	36 hours

Collaborators and Investigators

• Sponsor: Pulmatrix Inc.
• Collaborators: Celerion
• Investigators: Principal Investigator: Stephen Smith, MD, Celerion

Study record dates

Study Major Dates

• Study Start: February 1, 2012
• Primary Completion (Actual): December 1, 2012
• Study Completion (Actual): December 1, 2012

Study Registration Dates

• First Submitted: February 27, 2012
• First Submitted That Met QC Criteria: March 1, 2012
• First Posted (Estimate): March 2, 2012

Study Record Updates

• Last Update Posted (Estimate): July 19, 2013
• Last Update Submitted That Met QC Criteria: July 18, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Keywords: Fibrosis; Respiratory Tract Diseases; Lung Diseases
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 601-0009

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No