A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of ELX/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Enrollment
- 64
- Locations
- 21
- Primary Endpoint
- Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Study Overview
Brief Summary
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 6 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
Exclusion Criteria
- •History of study drug intolerance in parent study
- •Other protocol defined Inclusion/Exclusion criteria may apply.
Arms & Interventions
ELX/TEZ/IVA
Participants greater than or equal to (≥) 6 years and less than (<) 12 years of age and weighing <30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
Intervention: ELX/TEZ/IVA (Drug)
ELX/TEZ/IVA
Participants greater than or equal to (≥) 6 years and less than (<) 12 years of age and weighing <30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
Intervention: IVA (Drug)
Outcomes
Primary Outcomes
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Baseline up to Week 196
Secondary Outcomes
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)(From Baseline up to Week 192)
- Absolute Change in Sweat Chloride (SwCl)(From Baseline up to Week 192)
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score(From Baseline up to Week 192)
- Absolute Change in Body Mass Index (BMI)(From Baseline up to Week 192)
- Absolute Change in BMI-for-age Z-score(From Baseline up to Week 192)
- Number of Participants With Pulmonary Exacerbations (PEx) for 106/107(From Baseline up to Week 192)
- Number of CF-related Hospitalizations for 106/107(From Baseline up to Week 192)
- Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)(From Baseline up to Week 192)
- Absolute Change in Weight(From Baseline up to Week 192)
- Absolute Change in Weight-for-age Z-score(From Baseline up to Week 192)
- Absolute Change in Height(From Baseline up to Week 192)
- Absolute Change in Height-for-age Z-score(From Baseline up to Week 192)