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Phase 3 Completed N=172 Treatment

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

Source: ClinicalTrials.gov NCT04362761 ↗
Enrolled (actual)
172
Serious AEs
15.3%
Results posted
Jul 2023
Primary outcomePrimary: 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
◆ Published Evidence
Established
71citations · ~12 / year
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
The Cochrane database of systematic reviews · 2020 · Open access · Likely link

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.

Linked Publications (2)

  • Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
    The Cochrane database of systematic reviews · 2020 · 71 citations · Open access · Likely link
  • Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
    The Cochrane database of systematic reviews · 2023 · 22 citations · Open access · Likely link

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

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) and the linked publication. 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. Informational only — not medical advice.

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