Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
Launched by UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · Feb 3, 2013
Trial Information
Current as of June 02, 2025
Completed
Keywords
ClinConnect Summary
Ivacaftor is a cystic fibrosis (CF)channel potentiator that is associated with decreased sweat chloride concentration, improved lung function, and improved weight gain. It is currently FDA approved for use only in CF patients with the G551D gating mutation. In vivo data suggest that ivacaftor may potentiate CF channels coded for by many other mutations associated with residual channel function, i.e., that some CF protein is present in the cell membrane of affected cells. This includes all of the 9 other known gating mutations, but ivacaftor also may be effective in CF patients with non-gati...
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Two mutations known to cause cystic fibrosis and a sweat chloride concentration greater than or equal to 55 mmol
- • Greater than or equal to 6 years of age
- Exclusion Criteria:
- • Homozygous F508del with a sweat chloride greater than 85 mmol
- • Taking medication known to interact with ivacaftor and chooses not to discontinue that medication
- • Is pregnant or planning to become pregnant during the study period
- • Less than 6 years of age
About University Of California, San Francisco
The University of California, San Francisco (UCSF) is a leading academic institution renowned for its commitment to advancing healthcare through innovative research and clinical trials. With a focus on translating scientific discoveries into effective therapies, UCSF collaborates with a diverse array of stakeholders, including healthcare professionals, industry partners, and patient communities. The university's research programs emphasize interdisciplinary approaches and leverage cutting-edge technology to address complex medical challenges. As a sponsor of clinical trials, UCSF is dedicated to maintaining the highest standards of ethical conduct and scientific rigor, ensuring the safety and well-being of participants while contributing to the broader medical knowledge base.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
San Francisco, California, United States
Patients applied
Trial Officials
Dennis W Nielson, MD, PhD
Principal Investigator
University of California, San Francisco
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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