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Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03278314
Expanded Access Status : Approved for marketing
First Posted : September 11, 2017
Last Update Posted : November 2, 2018
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: tezacaftor/ivacaftor

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

Intervention Details:
  • Drug: tezacaftor/ivacaftor
    orally administered TEZ/IVA combination therapy
    Other Name: TEZ/IVA

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  • Males and females, 12 years of age and older with confirmed diagnosis of CF.

    1. - completed Vertex clinical studies NCT02565914 or NCT03150719, or
    2. - are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks from start of treatment because of the onset of respiratory AEs considered to be treatment related or (2) cannot initiate Orkambi because of chronic treatment with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow therapeutic index AND who meet at least 1 of the following criteria:

      • the highest percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 in the 6 months before the date of completion of the request form, or
      • documentation of being active on a lung transplant waiting list or documentation of being evaluated for lung transplantation, but deemed unsuitable because of contraindications, or
      • rapid and persistent loss of lung function, defined as at least a 20% relative decrease in ppFEV1 in the last 6 months and sustained for at least 1 month despite appropriate treatment.

Exclusion Criteria:

  • History of any comorbidity that, in the opinion of the treating physician, might pose undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history of advanced liver disease).
  • Ongoing or prior participation in an investigational drug study (with the exception of Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the previous investigational study drug or 30 days, whichever is longer, of first administration of TEZ/IVA.
  • Subjects who are pregnant.
  • Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or other CFTR modulator therapies.

Other protocol defined Inclusion/Exclusion criteria may apply.

No Contacts or Locations Provided
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Responsible Party: Vertex Pharmaceuticals Incorporated Identifier: NCT03278314    
Other Study ID Numbers: VX17-661-901
First Posted: September 11, 2017    Key Record Dates
Last Update Posted: November 2, 2018
Last Verified: October 2018
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action