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NCT04353817

A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes

Completed Phase 3 Results posted Last updated 26 July 2022
What this trial tests

Phase 3 trial testing ELX/TEZ/IVA in Cystic Fibrosis in 121 participants. Completed in 17 May 2021.

Timeline
19 June 2020
Primary endpoint
17 May 2021
17 May 2021

Quick facts

Lead sponsorVertex Pharmaceuticals Incorporated
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingquadruple
Primary purposetreatment
Enrollment121
Start date19 June 2020
Primary completion17 May 2021
Estimated completion17 May 2021
Sites34 locations across Denmark, France, Netherlands, United Kingdom, Germany, Israel, Canada, Switzerland

Drugs / interventions tested

Conditions studied

Sponsor

Vertex Pharmaceuticals Incorporated — full company profile →

Who can join

Adults 6 to 11, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Primary · From Baseline Through Week 24

The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.

GroupValue95% CI
Placebo-0.02± 0.16
ELX/TEZ/IVA-2.29± 0.16
Absolute Change in Sweat Chloride (SwCl) Secondary · From Baseline Through Week 24

Sweat samples were collected using an approved collection device.

GroupValue95% CI
Placebo-0.9± 1.5
ELX/TEZ/IVA-52.1± 1.5
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) Secondary · Day 1 up to Week 28
Participants With TEAEs
GroupValue95% CI
Placebo57
ELX/TEZ/IVA48
Participants With SAEs
GroupValue95% CI
Placebo9
ELX/TEZ/IVA4

Adverse events — posted to ClinicalTrials.gov

Time frame: Day 1 up to Week 28. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Placebo
Serious: 9/61 (15%)
Deaths: 0/61
ELX/TEZ/IVA
Serious: 4/60 (7%)
Deaths: 0/60

Serious adverse events (11 terms)

ReactionSystemPlaceboELX/TEZ/IVA
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations——
LymphadenitisBlood and lymphatic system disorders——
PhimosisCongenital, familial and genetic disorders——
Distal intestinal obstruction syndromeGastrointestinal disorders——
IntussusceptionGastrointestinal disorders——
General physical health deteriorationGeneral disorders——
Pneumonia pseudomonalInfections and infestations——
Varicella zoster virus infectionInfections and infestations——
Bacterial test positiveInvestigations——
Nasal polypsRespiratory, thoracic and mediastinal disorders——
RashSkin and subcutaneous tissue disorders——
Other adverse events (26 terms — click to expand)

ReactionSystemPlaceboELX/TEZ/IVA
CoughRespiratory, thoracic and mediastinal disorders——
HeadacheNervous system disorders——
Abdominal painGastrointestinal disorders——
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations——
Oropharyngeal painRespiratory, thoracic and mediastinal disorders——
NasopharyngitisInfections and infestations——
Productive coughRespiratory, thoracic and mediastinal disorders——
RhinorrhoeaRespiratory, thoracic and mediastinal disorders——
DiarrhoeaGastrointestinal disorders——
RashSkin and subcutaneous tissue disorders——
Abdominal pain upperGastrointestinal disorders——
NauseaGastrointestinal disorders——
FatigueGeneral disorders——
RhinitisInfections and infestations——
Upper respiratory tract infectionInfections and infestations——
Alanine aminotransferase increasedInvestigations——
VomitingGastrointestinal disorders——
Bacterial test positiveInvestigations——
Forced expiratory volume decreasedInvestigations——
Staphylococcus test positiveInvestigations——
ArthralgiaMusculoskeletal and connective tissue disorders——
Nasal polypsRespiratory, thoracic and mediastinal disorders——
PruritusSkin and subcutaneous tissue disorders——
SteatorrhoeaGastrointestinal disorders——
Aspartate aminotransferase increasedInvestigations——
Nasal congestionRespiratory, thoracic and mediastinal disorders——

Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Lymphadenitis, Phimosis, Distal intestinal obstruction syndrome, Intussusception, General physical health deterioration, Pneumonia pseudomonal, Varicella zoster virus infection.

Data from ClinicalTrials.gov NCT04353817 adverse events section.

Sponsor's own description

This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for <i>F508del</i> and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.
    Mall MA, Brugha R, Gartner S, Legg J, et al · · 2022 · cited 109× · PMID 35816621 · DOI 10.1164/rccm.202202-0392oc
  2. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
    Southern KW, Murphy J, Sinha IP, Nevitt SJ. · · 2020 · cited 32× · PMID 33331662 · DOI 10.1002/14651858.cd010966.pub3
  3. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
    Heneghan M, Southern KW, Murphy J, Sinha IP, et al · · 2023 · cited 10× · PMID 37983082 · DOI 10.1002/14651858.cd010966.pub4
  4. Analyzing the use and impact of elexacaftor/tezacaftor/ivacaftor on total cost of care and other health care resource utilization in a commercially insured population.
    Smith S, Borchardt M. · · 2022 · cited 8× · PMID 35737861 · DOI 10.18553/jmcp.2022.28.7.721
  5. Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.
    Silva Filho LVRFD, Athanazio RA, Tonon CR, Ferreira JC, et al · · 2024 · cited 3× · PMID 38198345 · DOI 10.36416/1806-3756/e20230187

Verify or expand the search:

Other trials of ELX/TEZ/IVA

Trials testing the same drug.

Other recruiting trials for Cystic Fibrosis

Currently open trials in the same condition.

Other Vertex Pharmaceuticals Incorporated trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing