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A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
Phase 3 trial testing ELX/TEZ/IVA in Cystic Fibrosis in 120 participants. Completed in 24 March 2023.
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 120 |
| Start date | 11 January 2021 |
| Primary completion | 24 March 2023 |
| Estimated completion | 24 March 2023 |
| Sites | 34 locations across Denmark, France, Netherlands, United Kingdom, Germany, Israel, Canada, Switzerland |
Vertex Pharmaceuticals Incorporated — full company profile →
6 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | 118 |
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | 13 |
Sweat samples were collected using an approved collection device.
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | -57.3 | ± 2.2 |
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | -57.5 | ± 2.3 |
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.
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | -1.74 | ± 0.18 |
| Group | Value | 95% CI |
|---|---|---|
| ELX/TEZ/IVA | -2.35 | ± 0.19 |
Time frame: Day 1 up to Week 100. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | ELX/TEZ/IVA |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — |
| Constipation | Gastrointestinal disorders | — |
| Enteritis | Gastrointestinal disorders | — |
| Ileus paralytic | Gastrointestinal disorders | — |
| Intestinal obstruction | Gastrointestinal disorders | — |
| Steatorrhoea | Gastrointestinal disorders | — |
| General physical health deterioration | General disorders | — |
| Bacterial disease carrier | Infections and infestations | — |
| Pneumonia pseudomonal | Infections and infestations | — |
| Pneumonia staphylococcal | Infections and infestations | — |
| Head injury | Injury, poisoning and procedural complications | — |
| Blood glucose increased | Investigations | — |
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | — |
| Weight gain poor | Metabolism and nutrition disorders | — |
| Haematuria | Renal and urinary disorders | — |
| Reaction | System | ELX/TEZ/IVA |
|---|---|---|
| COVID-19 | Infections and infestations | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — |
| Nasopharyngitis | Infections and infestations | — |
| Pyrexia | General disorders | — |
| Headache | Nervous system disorders | — |
| Upper respiratory tract infection | Infections and infestations | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — |
| Rhinitis | Infections and infestations | — |
| Abdominal pain | Gastrointestinal disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | — |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — |
| Productive cough | Respiratory, thoracic and mediastinal disorders | — |
| Diarrhoea | Gastrointestinal disorders | — |
| Viral upper respiratory tract infection | Infections and infestations | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — |
| SARS-CoV-2 test positive | Investigations | — |
| Abdominal pain upper | Gastrointestinal disorders | — |
| Influenza | Infections and infestations | — |
| Alanine aminotransferase increased | Investigations | — |
| Hordeolum | Infections and infestations | — |
| Nausea | Gastrointestinal disorders | — |
| Staphylococcus test positive | Investigations | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — |
| Ear pain | Ear and labyrinth disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Immunisation reaction | Immune system disorders | — |
| Bacterial disease carrier | Infections and infestations | — |
| Conjunctivitis allergic | Eye disorders | — |
| Bacterial test positive | Investigations | — |
| Fatigue | General disorders | — |
| Aspartate aminotransferase increased | Investigations | — |
| Decreased appetite | Metabolism and nutrition disorders | — |
Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Constipation, Enteritis, Ileus paralytic, Intestinal obstruction, Steatorrhoea, General physical health deterioration, Bacterial disease carrier.
Data from ClinicalTrials.gov NCT04545515 adverse events section.
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
3 peer-reviewed publications reference this trial (live from Europe PMC):
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