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

NCT04702360 | Unknown

• 1 other identifier: VX20-445-905
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

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

IVA monotablet for oral administration.

Also known as: ivacaftor, VX-770

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: all
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• Patients with severe hepatic impairment

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: January 6, 2021
• First Posted: January 8, 2021
• Last Updated: July 15, 2021

Record last verified: 2021-07