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Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

This study is currently recruiting participants.
Verified September 2012 by Vertex Pharmaceuticals Incorporated
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01685801
First received: September 6, 2012
Last updated: September 11, 2012
Last verified: September 2012
History of Changes
  Purpose

This study is a multiple within subject crossover study to evaluate the effect of ivacaftor on lung function in subjects aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function.


Condition Intervention Phase
Cystic Fibrosis
 Drug: ivacaftor
Drug: placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver)
Primary Purpose: Treatment
Official Title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted

Resource links provided by NLM:

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor
U.S. FDA Resources

Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Relative change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) after 2 weeks of treatment [ Time Frame: baseline and 2 weeks of treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in lung clearance index (LCI) after 2 weeks of treatment [ Time Frame: baseline and 2 weeks of treatment ] [ Designated as safety issue: No ]
  • Relative change from baseline in percent predicted FEV1 after 8 weeks of treatment [ Time Frame: baseline and 8 weeks of treatment ] [ Designated as safety issue: No ]
  • Change from baseline in LCI after 8 weeks of treatment [ Time Frame: baseline and 8 weeks of treatment ] [ Designated as safety issue: No ]
  • Change from baseline in sweat chloride after 8 weeks of treatment [ Time Frame: baseline and 8 weeks of treatment ] [ Designated as safety issue: No ]
  • Change from baseline in weight after 8 weeks of treatment [ Time Frame: baseline and 8 weeks of treatment ] [ Designated as safety issue: No ]
  • Safety, as determined by adverse events and vital signs [ Time Frame: baseline through 28 weeks (follow-up visit) ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 18
Study Start Date: September 2012
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sequence 1
ivacaftor > placebo > ivacaftor > placebo > ivacaftor (open-label period)
 Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours
Experimental: Sequence 2
ivacaftor > placebo > placebo > ivacaftor > ivacaftor (open-label period)
 Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours
Experimental: Sequence 3
placebo > ivacaftor > ivacaftor > placebo > ivacaftor (open-label period)
 Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours
Experimental: Sequence 4
placebo > ivacaftor > placebo > ivacaftor > ivacaftor (open-label period)
 Drug: ivacaftor
150 mg tablet for oral use, administered every 12 hours
Other Name: Tradename: KALYDECO
Drug: placebo
tablet for oral use, administered every 12 hours

Detailed Description:

CFTR Mutations associated with residual CFTR function or defective mRNA splicing include the following:

R117H, E56K, P67L, D110E, D110H, R117C, R347H, R352Q, A455E, D579G, S945L, L206W, R1070W, F1074L, D1152H, S1235R, D1270N, 2789+5G->A, 3849+10kbC->T, 3272-26A->G, 711+5G->A, 3120G->A, 1811+1.6kbA->G, 711+3A->G, 1898+3A->G, 1898+1G->A, 1717-1G->A, 1717-8G->A, 1342-2A->C, 405+3A->C, 1716G/A 1811+1G->C, 1898+5G->T, 3850-3T->G, IVS14b+5G->A, 1898+1G->T, 4005+2T->C, 621+3A->G, 621+1G->T

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female subjects with confirmed diagnosis of CF
  • Clinical evidence of residual CFTR function based on any 1 of the following: 1)Clinically documented residual exocrine pancreatic function, 2)Sweat chloride value ≤80 mmol/L at screening, or 3) Age of diagnosis ≥12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing
  • FEV1 ≥40%
  • 12 years of age or older
  • Willing to agree to meet the contraception requirements
  • Able to swallow tablets

Exclusion Criteria:

  • A copy of any of the following CFTR mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
  • Unable to perform spirometry
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01685801

Contacts
Contact: Medical Monitor 617.444.6777 medicalinfo@vrtx.com

Locations
United States, Colorado
Recruiting
Denver, Colorado, United States
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Investigators
Principal Investigator: Jerry Nick, MD National Jewish Health
  More Information

No publications provided

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01685801     History of Changes
Other Study ID Numbers: VX12-770-113
Study First Received: September 6, 2012
Last Updated: September 11, 2012
Health Authority: United States: Food and Drug Administration

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