Study of VX-770 in Cystic Fibrosis Subjects

• Register: ANZCTR
• Last refreshed on: 13 January 2020
• Main ID: ACTRN12610000491066
• Date of registration: 15/06/2010
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceutical Incorporated
• Public title: Study of VX-770 in Cystic Fibrosis Subjects
• Scientific title: An Open-Label, Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis.
• Date of first enrolment: 01/07/2010
• Target sample size: 200
• Recruitment status: Active, not recruiting
• URL: https://anzctr.org.au/ACTRN12610000491066.aspx
• Study type: Interventional
• Study design: Purpose: Treatment; Allocation: Non-randomised trial; Masking: Open (masking not used);Assignment: Single group;Type of endpoint: Safety/efficacy;
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Czech Republic, France, Germany, Ireland, United Kingdom, United States of America

Contacts

• Name: Medical Monitor

• Name: Medical Monitor

Key inclusion & exclusion criteria

Inclusion criteria: 1. Subjects who have completed the assigned study treatment Study 102 or Study 103. 2. Subjects who are female of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770) 3. Subjects who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator. 4. Subjects of childbearing potential and who are sexually active must meet the contraception requirements. 5. Subjects must sign the informed consent form (ICF), and where appropriate, assent must be obtained.
Exclusion criteria: 1. Subjects with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
2. Subjects with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject.
3. Subjects who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements.
4. Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications (e.g., St John's Wort) and grapefruit/grapefruit juice.

Age minimum: 6 Years
Age maximum: No limit
Gender: Both males and females

Health Condition(s) or Problem(s) studied

Cystic Fibrosis;
Cystic Fibrosis

Human Genetics and Inherited Disorders - Cystic fibrosis

Intervention(s)

VX-770 150mg, oral tablets, twice per day, for the sooner of approximately 96 weeks or until VX 770 is commercially available in each respective country

Primary Outcome(s)

To evaluate the safety of long-term VX-770 treatment in subjects with Cystic Fibrosis.as assessed by standard digital electrocardiogram(ECG), blood analysis, coagulation studies, urinalysis, vital signs, physical examination and patient reporting of any adverse event (e.g. gastrointestinal disturbance, nausea, cough, sinus pain, sore throat, etc.)[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Secondary Outcome(s)

Rate of change in weight, measured using calibrated scales.[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Absolute change from Day 1 of study VX08-770-105 (Study 105, current study) in FEV1, measured by spirometry.[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Change from Day 1 of Study 105 (current study) in Cystic Fibrosis Questionnaire-Revised (CFQ-R)[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Change from Day 1 of previous VX-770 study in CFQ-R[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Absolute change from Day 1 of previous VX-770 study in FEV1, measured by spirometry.
[Study 102, NCT00909532]
[Study 103, NCT00909727][After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Patients must commence this current trial upon completion of the last day on the initial trial.]

Pulmonary exacerbation, defined as new or change in antibiotic therapy for sinopulmonary signs/sypmtoms.
Assessed through sinopulmonary assessment and review of concomitant medication.[Throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Rate of decline in percent predicted Forced Expiratory Volume in 1 second (FEV1), measured by spirometry.[After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.]

Secondary ID(s)

ClinicalTrials.gov: NCT01117012

Source(s) of Monetary Support

Vertex Pharmaceutical Incorporated

Secondary Sponsor(s)

Omnicare Clinical Research Pty Ltd