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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by University of California, San Francisco
Sponsor:
Information provided by (Responsible Party):
University of California, San Francisco
ClinicalTrials.gov Identifier:
NCT01784419
First received: February 3, 2013
Last updated: January 22, 2014
Last verified: January 2014
  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:


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: October 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 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 placebo-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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01784419

Contacts
Contact: Jane Ye 415-502-2880 Jane.Ye@ucsf.edu
Contact: Dennis W Nielson, MD, PhD 415-476-3471 nielsond@peds.ucsf.edu

Locations
United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Jane Ye    415-502-2880    Jane.Ye@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
University of California, San Francisco
Investigators
Principal Investigator: Dennis W Nielson, MD, PhD University of California, San Francisco
  More Information

No publications provided

Responsible Party: 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: January 22, 2014
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 July 24, 2014