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

NCT ID: NCT01543191

Last Updated: 2013-07-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-02-29
• Study Completion Date: 2012-12-31

Brief Summary

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

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

PUR118

Group Type: EXPERIMENTAL

PUR118

Intervention Type: DRUG

low dose and high dose for 3 doses, BID

Placebo

Group Type: PLACEBO_COMPARATOR

PUR118

Intervention Type: DRUG

low dose and high dose for 3 doses, BID

Interventions

PUR118

low dose and high dose for 3 doses, BID

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celerion

INDUSTRY

Sponsor Role: collaborator

Pulmatrix Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Stephen Smith, MD

Role: PRINCIPAL_INVESTIGATOR

Celerion

Locations

Celerion

Belfast, Northern Ireland, United Kingdom

Countries

United Kingdom

Other Identifiers

601-0009

Identifier Type: -

Identifier Source: org_study_id