ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
July 8, 2021 updated by: Vertex Pharmaceuticals Incorporated
ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation
The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician requests.
Study Overview
Status
Approved for marketing
Conditions
Intervention / Treatment
Study Type
Expanded Access
Expanded Access Type
- Treatment IND/Protocol
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years to 11 years (Child)
Accepts Healthy Volunteers
N/A
Description
Key Inclusion Criteria:
- Patients with confirmed diagnosis of CF who have an F/any genotype
- Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment)
- Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) <40 for a minimum of 60 consecutive days before the date of completion of the request form
Key Exclusion Criteria:
- Patients with severe hepatic impairment
Other protocol-defined inclusion/exclusion criteria may apply
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
January 6, 2021
First Submitted That Met QC Criteria
January 6, 2021
First Posted (Actual)
January 8, 2021
Study Record Updates
Last Update Posted (Actual)
July 15, 2021
Last Update Submitted That Met QC Criteria
July 8, 2021
Last Verified
July 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Genetic Diseases, Inborn
- Respiratory Tract Diseases
- Digestive System Diseases
- Lung Diseases
- Infant, Newborn, Diseases
- Pancreatic Diseases
- Fibrosis
- Cystic Fibrosis
- Molecular Mechanisms of Pharmacological Action
- Membrane Transport Modulators
- Chloride Channel Agonists
- Elexacaftor
- Ivacaftor
Other Study ID Numbers
- VX20-445-905
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.