Clinical trial NCT01543191: A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
Official title: A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cystic Fibrosis
Keywords: Lung Diseases
Respiratory Tract Diseases
Fibrosis
Lead sponsor: Pulmatrix Inc.
Sponsor class: INDUSTRY
Collaborators: name: Celerion
First posted: 2012-03-02
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Safety and Tolerability
timeFrame: 4-6 weeks
Secondary outcomes:
measure: Pharmacodynamics
timeFrame: 36 hours
Other outcomes:
Locations:
city: Belfast
country: United Kingdom
facility: Celerion
state: Northern Ireland
Study officials:
affiliation: Celerion
name: Stephen Smith, MD
role: PRINCIPAL_INVESTIGATOR
References: