Study of long-term VX-770 treatment in Cystic Fibrosis subjects Age 6 and Older

• Register: EUCTR
• Last refreshed on: 14 March 2016
• Main ID: EUCTR2009-012997-11-CZ
• Date of registration: 12/05/2010
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of long-term VX-770 treatment in Cystic Fibrosis subjects Age 6 and Older
• Scientific title: An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis - PERSIST
• Date of first enrolment: 22/06/2010
• Target sample size: 217
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-012997-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 1

Countries of recruitment

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

Contacts

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion criteria:
- Subjects who have completed the assigned study treatment Study 102 or Study 103 who meet certain criteria.
- Subjects who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770).
- 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.
- Subjects of child bearing potential and who are sexually active must meet the contraception requirements.
- Subjects must sign the informed consent form (ICF), and where appropriate, assent must be obtained.

Are the trial subjects under 18? yes
Number of subjects for this age range: 73
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 118
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- 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.
- 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.
- Subjects who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements.
- Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications (e.g., St. John’s Wort) and grapefruit/grapefruit juice.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Cystic Fibrosis
MedDRA version: 16.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Kalydeco
Product Code: VX-770, VRT-813077
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: ivacaftor
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Other descriptive name: VRT-813077
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Main Objective: To evaluate the safety of long-term VX-770 treatment in subjects with cystic fibrosis (CF)

Secondary Objective: To evaluate the efficacy of long-term VX-770 treatment in subjects with CF

Primary end point(s): Safety as determined by adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital ECGs, vital signs, and physical examinations

Timepoint(s) of evaluation of this end point: Visits 1 to 5 (Day 1, Day 15, Week 24, Week 48, Week 78, Week 60,
Week 89, Week 96, Week 108, Week 120, Week 132, Week 144, Week 156, Week 168, Week 180, and Week 192

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Visits 1 to 5 (Day 1, Day 15, Week 24, Week 48, Week 78, Week 60,
Week 89, Week 96, Week 108, Week 120, Week 132, Week 144, Week 156, Week 168, Week 180, and Week 192

Secondary end point(s): Efficacy as determined by:
• Rate of decline in percent predicted FEV1
• Absolute change from baseline of Study 105 in FEV1
• Absolute change from baseline of previous VX-770 study in FEV1
• Change from baseline of Study 105 in Cystic Fibrosis Questionnaire-
Revised (CFQ-R) respiratory domain score
• Change from baseline of previous VX-770 study in CFQ-R respiratory domain score
• Pulmonary exacerbations (as defined in Section 12.3.6.1)
• Change from baseline of Study 105 in weight
• Change from baseline of previous VX-770 study in weight

Secondary ID(s)

NCT01117012

VX08-770-105

2009-012997-11-FR

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated