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Study Name: Hydroxychloroquine for the Treatment of CF Lung Disease
Study Type: Interventional
Intervention Category: Anti-Inflammatory
Study Sponsor: Perkett, Elizabeth

Funding for this trial has been provided in full or in part by Cystic Fibrosis Foundation Therapeutics, Inc.
Study Phase: Other
Recruitment Status: Trial Completed
Study Drug(s): Hydroxychloroquine
Number of Participants Being Recruited: 20
Single / Multi-Center: Single Center
Cystic Fibrosis (CF) remains a fatal disease.  Improvements in therapies have slowed the rate of decline and many of these therapies are aimed at decreasing inflammation.  Unfortunately anti inflammatory therapies including steroids and ibuprofen have 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.  In clinical medicine, chloroquine has been replaced primarily by hydroxychloroquine.   The most common use for hydroxychloroquine is for the treatment of malaria, but it is also used in chronic inflammatory disease such as arthritis and lupus.   Hydroxcychloroqine has also been used chronic pulmonary diseases but it has never been examined in CF.   This pilot study sought to determine whether there was any evidence that hydroxychloroquine has an anti inflammatory effect in CF.  Inflammatory markers were measured in sputum collected  from 20 CF patients before and after 4 weeks of hydroxychloroquine.
Age: 16 Years - 65 Years
FEV1: >= 40 Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Note: Detailed eligibility criteria information may be available on If a specific trial listing for this trial is available, a link to the specific listing will be present in the "More Information" section below.
Sponsor Contact Information: Perkett, Elizabeth
(615) 343-7617
Trial Specific Link on
Clinical Research Terms Glossary: Click here
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 (cell counts or cytokines).

Secondary Efficacy:

Not applicable


Two patients discontinued the study due to adverse events (one with GI symptoms and the other due to pulmonary exacerbation). 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 CF Conference (2008). These data may be preliminary and have not been peer-reviewed.


Not Available
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