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

Vertex Pharmaceuticals

Status

Conditions: Cystic Fibrosis

Treatments: Drug: ELX/TEZ/IVA; Drug: IVA

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT04702360

VX20-445-905

Details and patient eligibility

About

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.

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• 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

Key Exclusion Criteria:

• Patients with severe hepatic impairment

Other protocol-defined inclusion/exclusion criteria may apply