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Phase 3 N=172 Treatment

A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

Cystic Fibrosis

Enrolled (actual)
172
Serious AEs
15.3%
Results posted
Jul 2023
Primary outcome: Primary: Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 160; 26 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
ELX/TEZ/IVA (Drug); IVA (Drug)
Age
Pediatric, Adult, Older Adult · 12+ yrs
Sex
All
Sponsor
Vertex Pharmaceuticals Incorporated
Primary completion
Dec 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
160; 26
PRIMARY
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
50; 8

Summary

This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.

Eligibility Criteria

Key Inclusion Criteria

  • Completed study drug treatment in parent study (VX18-445-109); 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

Key Exclusion Criteria

  • History of study drug intolerance in parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04362761). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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