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

This study has been completed.
Sponsor:
Collaborator:
Celerion
Information provided by (Responsible Party):
Pulmatrix Inc.
ClinicalTrials.gov Identifier:
NCT01543191
First received: February 27, 2012
Last updated: July 18, 2013
Last verified: July 2013
  Purpose

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


Condition Intervention Phase
Cystic Fibrosis
Drug: PUR118
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF

Resource links provided by NLM:


Further study details as provided by Pulmatrix Inc.:

Primary Outcome Measures:
  • Safety and Tolerability [ Time Frame: 4-6 weeks ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Pharmacodynamics [ Time Frame: 36 hours ] [ Designated as safety issue: No ]

    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.



Enrollment: 10
Study Start Date: February 2012
Study Completion Date: December 2012
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PUR118 Drug: PUR118
low dose and high dose for 3 doses, BID
Placebo Comparator: Placebo Drug: PUR118
low dose and high dose for 3 doses, BID

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01543191

Locations
United Kingdom
Celerion
Belfast, Northern Ireland, United Kingdom, BT9 6AD
Sponsors and Collaborators
Pulmatrix Inc.
Celerion
Investigators
Principal Investigator: Stephen Smith, MD Celerion
  More Information

No publications provided

Responsible Party: Pulmatrix Inc.
ClinicalTrials.gov Identifier: NCT01543191     History of Changes
Other Study ID Numbers: 601-0009
Study First Received: February 27, 2012
Last Updated: July 18, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Pulmatrix Inc.:
Lung Diseases
Respiratory Tract Diseases
Fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on November 27, 2014