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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)

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

Status
APPROVED_FOR_MARKETING
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT04058210
Enrollment
Unknown
Registered
2019-08-15
Start date
Unknown
Completion date
Unknown
Last updated
2019-11-21

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(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.

Interventions

DRUGELX/TEZ/IVA

Fixed-dose combination tablet for oral administration.

DRUGIVA

150-mg tablet for oral administration.

Sponsors

Vertex Pharmaceuticals Incorporated
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
12 Years to No maximum

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/

Outcome results

None listed

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