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

NCT01543191 | Completed Phase 1

• 1 other identifier: 601-0009
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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

Keywords

Lung Diseases; Respiratory Tract Diseases; Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Pharmacodynamics

  36 hours

Study Arms (2)

PUR118

EXPERIMENTAL

Drug: PUR118

Placebo

PLACEBO COMPARATOR

Drug: PUR118

Interventions

PUR118 DRUG

low dose and high dose for 3 doses, BID

PUR118; Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pulmatrix Inc.: lead
• Celerion: collaborator

Study Sites (1)

Celerion

Belfast, Northern Ireland, BT9 6AD, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis; Lung Diseases; Respiratory Tract Diseases; Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Stephen Smith, MD

  Celerion

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2012
• First Posted: March 2, 2012
• Study Start: February 1, 2012
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: July 19, 2013

Record last verified: 2013-07

Locations

GB (1)