Cff.org

Clinical Research
Information Sheet
Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) is the drug discovery and development arm of the Cystic Fibrosis Foundation.
Study Name:
Hydroxychloroquine for the Treatment of CF Lung Disease Study Type:
Intervention Category:
Study Sponsor:
Funding for this trial has been provided in full or in part by Cystic FibrosisFoundation Therapeutics, Inc.
Study Phase:
Recruitment Status:
Study Drug(s):
Number of Participants Being Recruited:
Single / Multi-Center:
STUDY BACKGROUND INFORMATION:
Cystic Fibrosis (CF) remains a fatal disease. Improvements in therapies have slowed the rate of decline and many of thesetherapies are aimed at decreasing inflammation. Unfortunately anti inflammatory therapies including steroids and ibuprofenhave side effects which limit their use. Laboratory studies have shown that chloroquine can improved CF cell function,including decreasing the binding of pseudomonas.  It is known whether chloroquine would have a similar action in vivo. Inclinical medicine, chloroquine has been replaced primarily by hydroxychloroquine.  The most common use forhydroxychloroquine is for the treatment of malaria, but it is also used in chronic inflammatory disease such as arthritis andlupus.  Hydroxcychloroqine has also been used chronic pulmonary diseases but it has never been examined in CF.  Thispilot study sought to determine whether there was any evidence that hydroxychloroquine has an anti inflammatory effect inCF. Inflammatory markers were measured in sputum collected from 20 CF patients before and after 4 weeks of hydroxychloroquine.
ELIGIBILITY
P. aeruginosa status:
B. cepacia status:
Other Primary Eligibility Requirements:
Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific trial listing for this trial is available,a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
FOR MORE INFORMATION:
Sponsor Contact Information:
(615) 343-7617elizabeth.a.perkett@vanderbilt.edu Trial Specific
Link on
ClinicalTrials.gov:
Clinical Research Terms Glossary:
). You also can find out about CF clinical trials and studies in your area
by calling the CF Foundation’s Clinical Trials Hotline at (877) 8CF-JOIN.
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TRIAL RESULTS:
Primary Efficacy:
Twenty patients received 4 weeks of once daily treatment with hydroxychloroquine. Seventeen patients completed the study.
There were no statistically significant differences in lung function, weight in markers of inflammation in induced sputum (cellcounts or cytokines). Secondary Efficacy:
Two patients discontinued the study due to adverse events (one with GI symptoms and the other due to pulmonaryexacerbation). There were no statistically significant differences in safety labs and the drug appeared to be well-tolerated. The summary of data provided here is from an abstract presented at the Cystic Fibrosis Foundation North American CFConference (2008). These data may be preliminary and have not been peer-reviewed. Citation:
). You also can find out about CF clinical trials and studies in your area
by calling the CF Foundation’s Clinical Trials Hotline at (877) 8CF-JOIN.

Source: http://www.cff.org/UploadedFiles/ClinicalResearchPDF/CR60.pdf