Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

X
Trial Profile

A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 12 Apr 2023

Price : $35 *
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs Ivacaftor/lumacaftor (Primary)
  • Indications Cystic fibrosis
  • Focus Adverse reactions; Pharmacokinetics; Registrational
  • Sponsors Vertex Pharmaceuticals
  • Most Recent Events

    • 10 Apr 2023 According to Vertex Pharmaceuticals media release, company today announced based on data from this trial, Health Canada as a partner of Access Consortium, has granted Marketing Authorization for the expanded use of PrORKAMBI (lumacaftor/ivacaftor) for the treatment of cystic fibrosis (CF) in 30 children ages 1 to <2 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
    • 21 Jan 2019 According to a media release, based on the data of this study, the European Commission has granted approval of the label extension for ORKAMBI (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease.
    • 13 Dec 2018 According to Vertex Pharmaceuticals media release, based on the data from this trial, Health Canada has granted Market Authorization for ORKAMBI (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del CFTR mutation.

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top