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Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

This study is not yet open for participant recruitment.
Verified February 2013 by University of California, San Francisco
Sponsor:
Information provided by (Responsible Party):
Dennis Nielson, University of California, San Francisco
ClinicalTrials.gov Identifier:
NCT01784419
First received: February 3, 2013
Last updated: NA
Last verified: February 2013
History: No changes posted
  Purpose

This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. However, it is highly likely that CF patients with many other mutations can benefit similarly from this drug, some of whom can be identified by phenotype or genotype.

We will enroll up to 30 CF subjects with clinical presentations in which there is one or more signs of residual CF channel function. The signs of residual function include: normal digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF disease in a CF patient with severe gene mutations. The primary outcome measure will be the difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor. Secondary outcome measured will be lung function.


Condition Intervention
Cystic Fibrosis
 Drug: ivacaftor

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Screening
Official Title: Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

Resource links provided by NLM:

Drug Information available for: Ivacaftor
U.S. FDA Resources

Further study details as provided by University of California, San Francisco:

Primary Outcome Measures:
  • Sweat chloride concentration [ Time Frame: 14 +/- 2 days ] [ Designated as safety issue: No ]
    Sweat chloride concentration measured by pilocarpine iontophoresis, a standard clinical laboratory technique. Sweat collection accomplished with the Wescor Macroduct system. Sweat chloride is measured at the start and end of each study period. There are two study periods during which subjects take either ivacaftor or placebo.


Secondary Outcome Measures:
  • Spirometry [ Time Frame: 14 +/- 2 days ] [ Designated as safety issue: No ]
    Standard spirometry will be performed at the start and end of each 2 week study period. Subjects will take study drug (ivacaftor or placebo during each study period.

  • Multibreath washout testing [ Time Frame: 14 +/- 2 days ] [ Designated as safety issue: No ]
    Subjects will perform multibreath washout testing using standard techniques to measure functional residual capacity and lung clearance index at the beginning and end of each study period.


Estimated Enrollment: 30
Study Start Date: May 2013
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ivacaftor-placebo
The ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course.
 Drug: ivacaftor
Prospective randomized double-blinded placebo controlled crossover study of the effects of ivacaftor in selected cystic fibrosis patients
Other Names:
  • Kalydeco
  • VX-770
Experimental: placebo-ivacaftor
The p;acebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
 Drug: ivacaftor
Prospective randomized double-blinded placebo controlled crossover study of the effects of ivacaftor in selected cystic fibrosis patients
Other Names:
  • Kalydeco
  • VX-770

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Two mutations known to cause cystic fibrosis and a sweat chloride concentration 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01784419

Contacts
Contact: Dennis W Nielson, MD, PhD 415-476-3471 nielsond@peds.ucsf.edu
Contact: Meghan McGarry, MD 415-476-9278 Meghan.McGarry@ucsf.edu

Locations
United States, California
University of California, San Francisco Not yet recruiting
San Francisco, California, United States, 94143
Contact: Meghan McGarry, MD     415-476-9278     Meghan.McGarry@ucsf.edu    
Contact: Dennis W Nielson, MD. PhD     415-476-3471     nielsond@peds.ucsf.edu    
Principal Investigator: Dennis W Nielson, MD, PhD            
Sub-Investigator: Meghan McGarry, MD            
Sub-Investigator: Ngoc Ly, MD, MPH            
Sub-Investigator: Mary Ellen Kleinhenz, MD            
Sponsors and Collaborators
Dennis Nielson
Investigators
Principal Investigator: Dennis W Nielson, MD, PhD University of California, San Francisco
  More Information

No publications provided

Responsible Party: Dennis Nielson, Professor of Pediatrics, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT01784419     History of Changes
Other Study ID Numbers: FS-001
Study First Received: February 3, 2013
Last Updated: February 3, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of California, San Francisco:
Cystic fibrosis
Ivacaftor
Genotype
 Phenotype
Sweat chloride
Pancreatic sufficient

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
 Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 16, 2013