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NCT04058210 Clinical Trial

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

Cystic Fibrosis Clinical Trial

Official title:
VX-445/TEZ/IVA Triple Combination Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function CFTR Mutation

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04058210
Other study ID # VX18-445-901
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date November 2019
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.

Recruitment information / eligibility
Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 12 Years and older
Eligibility 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

Related Conditions & MeSH terms

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration.
• IVA
150-mg tablet for oral administration.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated
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