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Restore CFTR Protein Completed with Results

VX-770 Expanded Access Program (Vertex VX11-770-901)

This open-label expanded access program (EAP) study was designed to provide ivacaftor to patients in critical medical need prior to commercial product availability.

 

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    Less than 40%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Have the G551D-CFTR mutation in at least one allele. Clarification of FEV1 requirement: Highest FEV1 in the 6 months prior to screening is <=40% of predicted value or patient is documented to be active on the lung transplant wait list.

Study Results

  • What We Learned:

  • Primary Findings:

    Effectiveness:

    This program was available between July 2011 and March 2012. A total of 44 participants received ivacaftor (150 mg every 12 h).

     

    At 24 weeks of treatment with ivacaftor, improvement in lung function (mean absolute increase in FEV1 % predicted of 5.5%) and a 3.3 kg mean increase in weight from baseline was observed.

    Safety:

    The primary endpoint was safety as determined by adverse events. Ivacaftor was well tolerated with the most frequently reported AEs consistent with those expected in patients with advanced CF lung disease. Pulmonary exacerbation was the most commonly reported AE (43.2%). The rate of serious adverse events was consistent with disease severity.

  • Citation:

    J Cyst Fibros ;DOI 10.1016/j.jcf.2015.01.008

For current information about the overall development status of this drug, please check the Drug Development Pipeline.

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    No

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    9 months

  • Number of Study Visits:

    1

Additional Information

  • Phase: ?more info

    Not Applicable

  • Study Sponsor: ?more info

    Vertex

  • Study Drugs:

    Ivacaftor (Kalydeco)

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    6 Years and Older

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    Less than 40%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Have the G551D-CFTR mutation in at least one allele. Clarification of FEV1 requirement: Highest FEV1 in the 6 months prior to screening is <=40% of predicted value or patient is documented to be active on the lung transplant wait list.

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