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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01543191
• Other study ID #: 601-0009
• Status: Completed
• Phase: Phase 1
• First received: February 27, 2012
• Last updated: July 18, 2013
• Start date: February 2012
• Est. completion date: December 2012

Study information

• Verified date: July 2013
• Source: Pulmatrix Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: December 2012
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 55 Years

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• PUR118
low dose and high dose for 3 doses, BID

Locations

Country	Name	City	State
United Kingdom	Celerion	Belfast	Northern Ireland

Sponsors (2)

Lead Sponsor	Collaborator
Pulmatrix Inc.	Celerion

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	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	No