Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03278314
• Date of registration: 06/09/2017
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis
• Scientific title: Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis
• Date of first enrolment: August 27, 2017
• Recruitment status: Approved for marketing
• URL: https://clinicaltrials.gov/show/NCT03278314
• Study type: Expanded Access

Key inclusion & exclusion criteria

Inclusion Criteria:

- Males and females, 12 years of age and older with confirmed diagnosis of CF.

1. - completed Vertex clinical studies NCT02565914 or NCT03150719, or

2. - are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks
from start of treatment because of the onset of respiratory AEs considered to be
treatment related or (2) cannot initiate Orkambi because of chronic treatment
with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow
therapeutic index AND who meet at least 1 of the following criteria:

- the highest percent predicted forced expiratory volume in 1 second (ppFEV1)
is <40 in the 6 months before the date of completion of the request form, 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, or

- rapid and persistent loss of lung function, defined as at least a 20%
relative decrease in ppFEV1 in the last 6 months and sustained for at least
1 month despite appropriate treatment.

Exclusion Criteria:

- History of any comorbidity that, in the opinion of the treating physician, might pose
undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history
of advanced liver disease).

- Ongoing or prior participation in an investigational drug study (with the exception of
Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the
previous investigational study drug or 30 days, whichever is longer, of first
administration of TEZ/IVA.

- Subjects who are pregnant.

- Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or
other CFTR modulator therapies.

Other protocol defined Inclusion/Exclusion criteria may apply.



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: tezacaftor/ivacaftor

Secondary ID(s)

VX17-661-901

Source(s) of Monetary Support

Please refer to primary and secondary sponsors