Rollover Study of VX-770 in Cystic Fibrosis Subjects

Cystic Fibrosis Clinical Trial

Official title:

An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01117012
• Other study ID #: VX08-770-105
• Secondary ID: PERSIST
• Status: Completed
• Phase: Phase 3
• First received: May 3, 2010
• Last updated: June 12, 2015
• Start date: July 2010
• Est. completion date: May 2014

Study information

• Verified date: June 2015
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyIreland: Irish Medicines BoardFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Federal Institute for Drugs and Medical DevicesCzech Republic: State Institute for Drug ControlAustralia: Department of Health and Ageing Therapeutic Goods AdministrationCanada: Health Canada
• Study type: Interventional

Clinical Trial Summary

The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.

Description:

This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 192
• Est. completion date: May 2014
• Est. primary completion date: May 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

1. Participants who have completed the assigned study treatment in Study 102 or Study 103

2. Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)

3. Participants 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. Participants of child bearing potential and who are sexually active must meet the contraception requirements

5. Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained

Exclusion Criteria:

1. Participants 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. Participants 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. Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements

4. Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Ivacaftor

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated	Cystic Fibrosis Foundation Therapeutics

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)	Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.	Study 105: Day 1 up to Week 168	Yes
Secondary	Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.	Study 105: Baseline through Week 96	No
Secondary	Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.	Study 105: Baseline through Week 96	No
Secondary	Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96	The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.	Study 105: Baseline through Week 96	No
Secondary	Annualized Pulmonary Exacerbation Event Rate	Annualized event rate was calculated by regression with negative binomial distribution.	Study 105: Day 1 through Week 96	No
Secondary	Annualized Duration of Pulmonary Exacerbation Events		Study 105: Day 1 through Week 96	No
Secondary	Absolute Change From Study 105 Baseline in Weight Through Week 96	Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.	Study 105: Baseline through Week 96	No