VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

NCT ID: NCT04058210

Last Updated: 2019-11-21

Basic Information

• Recruitment Status: APPROVED_FOR_MARKETING
• Study Classification: EXPANDED_ACCESS

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.

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVA

Fixed-dose combination tablet for oral administration.

Intervention Type: DRUG

IVA

150-mg tablet for oral administration.

Intervention Type: DRUG

Other Intervention Names

elexacaftor/tezacaftor/ivacaftor; VX-445/VX-661/VX-770; ivacaftor; VX-770

Eligibility Criteria

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Other Identifiers

VX18-445-901

Identifier Type: -

Identifier Source: org_study_id