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

• Conditions: Cystic Fibrosis

• Registration Number: NCT04058210
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-08-13
• Study First Submitted QC: 2019-08-13
• Study First Posted: 2019-08-15
• Status Verified: 2019-11
• Last Update Submitted: 2019-11-19
• Last Update Posted: 2019-11-21

Recruitment & Eligibility

• Status: APPROVED_FOR_MARKETING
• Sex: All
• Target Recruitment: Not specified

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/Exclusion criteria may apply

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified