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A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
Phase 3 trial testing TEZ/IVA in Cystic Fibrosis in 130 participants. Completed in 29 September 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 | 130 |
| Start date | 25 April 2018 |
| Primary completion | 28 October 2020 |
| Estimated completion | 29 September 2023 |
| Sites | 55 locations across Denmark, France, Belgium, Ireland, United Kingdom, Germany, Poland, Canada |
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 |
|---|---|---|
| Part A: TEZ/IVA | 129 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | 31 |
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 |
|---|---|---|
| Part A: TEZ/IVA | -0.95 | -1.38 – -0.52 |
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 |
|---|---|---|
| Part A: TEZ/IVA | -2.04 | ± 1.73 |
Sweat samples were collected using an approved collection device.
| Group | Value | 95% CI |
|---|---|---|
| TEZ/IVA | -13.8 | -17.7 – -9.9 |
Sweat samples were collected using an approved collection device.
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | -16.2 | -21.9 – -10.5 |
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | 6.4 | 3.5 – 9.3 |
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | 6.0 | 1.1 – 10.8 |
BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2).
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | 1.25 | 1.00 – 1.49 |
BMI was defined as weight in kg divided by m\^2.
| Group | Value | 95% CI |
|---|---|---|
| Part A: TEZ/IVA | 1.19 | 0.74 – 1.64 |
| Group | Value | 95% CI |
|---|---|---|
| TEZ/IVA | 53 |
| Group | Value | 95% CI |
|---|---|---|
| TEZ/IVA | 8 |
Time frame: Day 1 Through Safety Follow-up Visit (up to Week 100 for Part A, and up to Week 192 for Part B). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Part A: TEZ/IVA | Part B: TEZ/IVA |
|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — |
| Bacterial test positive | Investigations | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | — | — |
| Immune thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | — | — |
| Intestinal obstruction | Gastrointestinal disorders | — | — |
| Small intestinal obstruction | Gastrointestinal disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Asthenia | General disorders | — | — |
| Pyrexia | General disorders | — | — |
| Bacterial disease carrier | Infections and infestations | — | — |
| Chronic sinusitis | Infections and infestations | — | — |
| Device related sepsis | Infections and infestations | — | — |
| Gastroenteritis | Infections and infestations | — | — |
| Influenza | Infections and infestations | — | — |
| Pertussis | Infections and infestations | — | — |
| Respiratory tract infection bacterial | Infections and infestations | — | — |
| Septic shock | Infections and infestations | — | — |
| Sinusitis | Infections and infestations | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — |
| Upper respiratory tract infection bacterial | Infections and infestations | — | — |
| Reaction | System | Part A: TEZ/IVA | Part B: TEZ/IVA |
|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — |
| Pyrexia | General disorders | — | — |
| Nasopharyngitis | Infections and infestations | — | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — |
| Productive cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Headache | Nervous system disorders | — | — |
| Bacterial test positive | Investigations | — | — |
| COVID-19 | Infections and infestations | — | — |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Influenza | Infections and infestations | — | — |
| Alanine aminotransferase increased | Investigations | — | — |
| Rhinitis | Infections and infestations | — | — |
| Pseudomonas test positive | Investigations | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Viral upper respiratory tract infection | Infections and infestations | — | — |
| Gastroenteritis | Infections and infestations | — | — |
| Ear pain | Ear and labyrinth disorders | — | — |
| Abdominal pain upper | Gastrointestinal disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Ear infection | Infections and infestations | — | — |
| Otitis media | Infections and infestations | — | — |
| Pharyngitis | Infections and infestations | — | — |
| Pharyngitis streptococcal | Infections and infestations | — | — |
| Aspartate aminotransferase increased | Investigations | — | — |
| Fatigue | General disorders | — | — |
| Bacterial disease carrier | Infections and infestations | — | — |
| Forced expiratory volume decreased | Investigations | — | — |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — |
| Tonsillitis | Infections and infestations | — | — |
| Staphylococcus test positive | Investigations | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Bacterial test positive, Abdominal pain, Nasal polyps, Immune thrombocytopenia, Cystic fibrosis related diabetes, Constipation, Diarrhoea.
Data from ClinicalTrials.gov NCT03537651 adverse events section.
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.
6 peer-reviewed publications reference this trial (live from Europe PMC):
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