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ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation

Status
APPROVED_FOR_MARKETING
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT04702360
Enrollment
Unknown
Registered
2021-01-08
Start date
Unknown
Completion date
Unknown
Last updated
2021-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

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.

Interventions

DRUGELX/TEZ/IVA

Fixed-dose combination tablet for oral administration.

DRUGIVA

IVA monotablet for oral administration.

Sponsors

Vertex Pharmaceuticals Incorporated
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
6 Years to 11 Years

Inclusion criteria

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/

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026