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

NCT04058210 | Unknown

• 1 other identifier: VX18-445-901
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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 DRUG

Fixed-dose combination tablet for oral administration.

Also known as: elexacaftor/tezacaftor/ivacaftor, VX-445/VX-661/VX-770

IVA DRUG

150-mg tablet for oral administration.

Also known as: ivacaftor, VX-770

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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

You may not qualify if:

• Patients with severe hepatic impairment (Child-Pugh Class C)
• 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
• Pregnancy

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination; ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 13, 2019
• First Posted: August 15, 2019
• Last Updated: November 21, 2019

Record last verified: 2019-11