Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03278314




Registration number
NCT03278314
Ethics application status
Date submitted
6/09/2017
Date registered
11/09/2017
Date last updated
2/11/2018

Titles & IDs
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
Secondary ID [1] 0 0
VX17-661-901
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Expanded Access
Description of intervention(s) / exposure
Treatment: Drugs - tezacaftor/ivacaftor

Treatment: Drugs: tezacaftor/ivacaftor
orally administered TEZ/IVA combination therapy
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key 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.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Key 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.

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Approved for marketing
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who
completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719).
To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of
age and older, homozygous for F508del.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03278314
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03278314