Repeatability and Response Study of Absorptive Clearance Scans

Cystic Fibrosis Clinical Trial

Official title:

Repeatability and Response Study of Absorptive Clearance Scans

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01887197
• Other study ID #: 1RO1 HL108929-01 (B)
• Status: Completed
• Phase: Phase 1
• Start date: June 2013
• Est. completion date: May 2018

Study information

• Verified date: June 2018
• Source: University of Pittsburgh
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The investigators are developing a new nuclear medicine imaging technique for measuring liquid absorption in the airways that can be applied to screen new medications being developed to treat cystic fibrosis (CF). The investigators believe that the absorption of the small molecule radiopharmaceutical Indium 111 diethylene triamine pentaacetic acid (In-DTPA) will indicate changes in liquid absorption in the airways and demonstrate whether new CF medications will be effective. In this study the investigators will determine whether the imaging technique will demonstrate similar results when it is repeated on different days. They will also determine how their results change when subjects utilize several common CF medications.

Description:

Cystic Fibrosis (CF) is an autosomal recessive genetic disease, caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) that impairs ion transport at epithelial surfaces. This results in the accumulation of dehydrated secretions in the airways and chronic infection and inflammation in the lungs, leading to significant morbidity and mortality. The investigators understanding of CF pathogenesis has increased substantially and many new targeted therapies are being developed to treat this disease, however, the measurements of clinical efficacy used to evaluate these therapies require long trials to demonstrate an effect. New translational techniques are needed to assess changes in the most basic aspects of the disease and allow for the rapid screening of disease-altering therapies. The investigators have recently developed a novel aerosol-based imaging technique to measure liquid absorption in the airways - a central pathophysiological process related to CFTR dysfunction. The investigators propose that airway liquid hyper-absorption is a key link between cellular defects in ion and fluid transport and progressive airway dysfunction in CF. Thus The investigators technique may provide a measure of disease severity and rapid indication of therapeutic correction in advance of currently available outcome measures.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: May 2018
• Est. primary completion date: December 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• subjects 18 years old or older with a diagnosis of cystic fibrosis as determined by sweat test or genotype and clinical symptoms who are clinically stable as determined by a physician co-investigator

Exclusion Criteria:

• one second forced expiratory volume (FEV1) <50% of predicted

• nursing mother

• positive urine pregnancy test or unwilling to test

• cigarette smoker

• unwilling to stop hypertonic saline therapy for 72 hours prior to each test day

• are intolerant to hypertonic saline (response only)

• are intolerant to any inhaled therapies (response only)

• fail mannitol tolerance testing (response only)

• have a history of excessive (uncontrollable) coughing after an osmotic stimulus (response only)

• have a history of hemoptysis (response only)

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Other:
• Absorptive clearance scan
Subjects inhale a nebulized mixture of the radiopharmaceuticals Indium 111-DTPA and Technetium 99m sulfur colloid.

Drug:
• inhaled hypertonic saline (7%)
nebulized hypertonic saline (7%)
• mannitol inhalation powder
mannitol inhalation powder

Locations

Country	Name	City	State
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Tim Corcoran

Country where clinical trial is conducted

United States, 

References & Publications (2)

Corcoran TE, Thomas KM, Brown S, Myerburg MM, Locke LW, Pilewski JM. Liquid hyper-absorption as a cause of increased DTPA clearance in the cystic fibrosis airway. EJNMMI Res. 2013 Feb 27;3(1):14. doi: 10.1186/2191-219X-3-14. — View Citation

Corcoran TE, Thomas KM, Myerburg MM, Muthukrishnan A, Weber L, Frizzell R, Pilewski JM. Absorptive clearance of DTPA as an aerosol-based biomarker in the cystic fibrosis airway. Eur Respir J. 2010 Apr;35(4):781-6. doi: 10.1183/09031936.00059009. Epub 2009 Aug 28. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	absorptive clearance variability	The difference in absorptive clearance measured on two different study days in the same subjects as an indicator of measurement variability	30 days
Primary	absorptive clearance response	The change in absorptive clearance demonstrated in response to therapy when compared to baseline	14 day

External Links

•   Dr. Corcoran's Research Page