ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT04702360
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
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 & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method