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VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

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ClinicalTrials.gov Identifier: NCT04058210
Expanded Access Status : Approved for marketing
First Posted : August 15, 2019
Last Update Posted : November 21, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation in response to unsolicited physician requests.

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: ELX/TEZ/IVA Drug: IVA

Study Design
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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: VX-445/TEZ/IVA Triple Combination Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function CFTR Mutation

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Interventions
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Intervention Details:
  • Drug: ELX/TEZ/IVA
    Fixed-dose combination tablet for oral administration.
    Other Names:
    • elexacaftor/tezacaftor/ivacaftor
    • VX-445/VX-661/VX-770
  • Drug: IVA
    150-mg tablet for oral administration.
    Other Names:
    • ivacaftor
    • VX-770
Eligibility Criteria
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Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

Inclusion Criteria:

1. Patients who have F/MF genotypes AND who meet at least 1 of the following criteria:

  • The percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 for a minimum of 2 months before the date of the request, 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

Exclusion Criteria:

  1. Patients with severe hepatic impairment (Child-Pugh Class C)
  2. History of any other comorbidity that, in the opinion of the treating physician, might pose undue risk in administering ELX/TEZ/IVA to the patient
  3. Pregnancy

Other protocol defined Inclusion/Exclusion criteria may apply

Contacts and Locations
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No Contacts or Locations Provided
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04058210    
Other Study ID Numbers: VX18-445-901
First Posted: August 15, 2019    Key Record Dates
Last Update Posted: November 21, 2019
Last Verified: November 2019
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Elexacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action