GIST: Using Rheological Methods to Characterize Cystic Fibrosis (CF) Sputum and the Effects of Mucoactive Agents

• Sponsor: University of California, San Francisco (Other)
• Overall Status: Completed
• CT.gov ID: NCT00758771
• Collaborator: Genentech, Inc. (Industry)
• Enrollment: 38
• Location: 1
• Duration (Months): 62
• Patients Per Site Per Month: 0.6

Study Details

Study Description

Brief Summary

The purpose of this study is to characterize the rheological properties of cystic fibrosis (CF) and healthy sputum and to examine the effects of mucoactive agents on the rheology of CF and healthy sputum. By collaborating with Genentech, the investigators (scientists at UCSF) plan to incorporate the latest scientific findings into our work to discover and develop new treatments for CF.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis

Detailed Description

There are two major mechanisms for mucus clearance in the airway, both of which are dependent upon optimal mucus viscosity and elasticity. These mechanisms are severely impaired in cystic fibrosis. The physical properties of sputum can be measured using rheological methods, enabling comparison between mucus in health and in disease. Therapies which enhance mucus clearance from the airway and decrease the volume of airway secretions are collectively called "mucoactive agents." Therapies which specifically disrupt innate mucus architecture by breaking intermolecular entanglements and bonds are called mucolytic agents. Mucolytic drugs can be considered in three general categories: classic mucolytics (n-acetylcysteine), peptide mucolytics (Pulmozyme®), and non-destructive mucolytics (hypertonic saline). Using state-of-the-art rheological methods, we can characterize the physical properties of CF mucus and measure the rheologic effects of mucoactive drugs more accurately and reproducibly than what has been previously done in the literature. In this way, we will determine which of the current mucoactive agents are most effective in normalizing sputum rheology in CF, and we will gain important insights about the limitations of current mucoactive drugs.

Study Design

• Study Type: Observational
• Actual Enrollment: 38 participants
• Observational Model: Case-Control
• Time Perspective: Prospective
• Official Title: Using State-of-the-art Rheological Methods to Characterize CF Sputum and the Effects of Mucoactive Agents: A Pilot Study
• Study Start Date: Oct 1, 2007
• Actual Primary Completion Date: Dec 1, 2012
• Actual Study Completion Date: Dec 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Cystic Fibrosis People who have been diagnosed with cystic fibrosis
Healthy People who do not have cystic fibrosis and who do not have any other lung conditions

Outcome Measures

Primary Outcome Measures

1. Shear Rheology [Cross-sectional]

   Baseline measure of sputum shear rheology

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Inclusion Criteria:

• Healthy control subjects:

• Age 18-65

• No history of lung disease or cardiac disease

• Cystic fibrosis subjects:

• Age 18-65

• Diagnosis of CF - sweat chloride values > 60 mM on pilocarpine iontophoresis sweat tests and/or 2 allelic CF-producing mutations by genetic analysis

Exclusion Criteria:

• Use of recreational drugs within 1 year prior to enrollment

• Use of tobacco within 1 year prior to enrollment, or > 10 pack-year tobacco history

• Upper respiratory tract infection in the 4 weeks prior to enrollment in the study

• Current use of antihistamines to treat allergies

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCSF Airway Clinical Research Center	San Francisco	California	United States	94143-0130

Sponsors and Collaborators

• University of California, San Francisco
• Genentech, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• UCSF Airway Clinical Research Center website

Publications

None provided.

• Responsible Party: University of California, San Francisco
• ClinicalTrials.gov Identifier: NCT00758771
• Other Study ID Numbers: 10-02116
• First Posted: Sep 25, 2008
• Last Update Posted: Mar 5, 2014
• Last Verified: Jan 1, 2014

• Additional relevant MeSH terms: Cystic Fibrosis; Fibrosis

Study Results

Participant Flow

Arm/Group Title	Cystic Fibrosis	Healthy
Arm/Group Description	Individuals who have been diagnosed with cystic fibrosis.	Individuals who do not have cystic fibrosis and who do not have any other lung conditions
Period Title: Overall Study
STARTED	18	20
COMPLETED	15	15
NOT COMPLETED	3	5

Baseline Characteristics

Arm/Group Title	Cystic Fibrosis	Healthy	Total
Arm/Group Description	Individuals who have been diagnosed with cystic fibrosis.	Individuals who do not have cystic fibrosis and who do not have any other lung conditions	Total of all reporting groups
Overall Participants	15	15	30
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	36.6 (12.8)	41.9 (10.8)	38.8 (11.9)
Sex: Female, Male (Count of Participants)
Female	4 26.7%	9 60%	13 43.3%
Male	11 73.3%	6 40%	17 56.7%
Region of Enrollment (participants) [Number]
United States	15 100%	15 100%	30 100%

Outcome Measures

1. Primary Outcome

Title	Shear Rheology
Description	Baseline measure of sputum shear rheology
Time Frame	Cross-sectional

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Cystic Fibrosis	Healthy
Arm/Group Description	Individuals who have been diagnosed with cystic fibrosis.	Individuals who do not have cystic fibrosis and who do not have any other lung conditions
Measure Participants	15	15
Mean (Standard Error) [Pascal]	6.0 (2.0)	1.8 (0.2)

Adverse Events

Arm/Group Title	Cystic Fibrosis		Healthy
Arm/Group Description	Individuals who have been diagnosed with cystic fibrosis.		Individuals who do not have cystic fibrosis and who do not have any other lung conditions
All Cause Mortality
	Cystic Fibrosis		Healthy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Cystic Fibrosis		Healthy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/15 (0%)		0/15 (0%)
Other (Not Including Serious) Adverse Events
	Cystic Fibrosis		Healthy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/15 (0%)		0/15 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.