Last reviewed · How we verify
A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
Phase 3 trial testing Tezacaftor/Ivacaftor in Cystic Fibrosis in 98 participants. Completed in 9 August 2018.
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 98 |
| Start date | 24 May 2017 |
| Primary completion | 9 August 2018 |
| Estimated completion | 9 August 2018 |
| Sites | 53 locations across France, United States, Germany |
Vertex Pharmaceuticals Incorporated — full company profile →
12 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.
RAESIs included chest discomfort, dyspnea (shortness of breath), respiration abnormal (chest tightness), asthma, bronchial hyperreactivity, bronchospasm, and wheezing.
| Group | Value | 95% CI |
|---|---|---|
| Placebo | 10 | |
| TEZ/IVA | 7 |
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Group | Value | 95% CI |
|---|---|---|
| Placebo | -0.6 | ± 3.4 |
| TEZ/IVA | 2.2 | ± 4.8 |
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Group | Value | 95% CI |
|---|---|---|
| Placebo | -1.5 | ± 8.1 |
| TEZ/IVA | 5.2 | ± 12.0 |
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. 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 |
|---|---|---|
| Placebo | 4.7 | ± 15.4 |
| TEZ/IVA | 5.7 | ± 14.2 |
| Group | Value | 95% CI |
|---|---|---|
| Placebo | 2 | |
| TEZ/IVA | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Placebo | 39 | |
| TEZ/IVA | 37 |
| Group | Value | 95% CI |
|---|---|---|
| Placebo | 9 | |
| TEZ/IVA | 5 |
Time frame: Day 1 up to Day 84. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Placebo | TEZ/IVA |
|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — |
| Sepsis | Infections and infestations | — | — |
| Lower respiratory tract infection | Infections and infestations | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Multiple organ dysfunction syndrome | General disorders | — | — |
| Suicidal ideation | Psychiatric disorders | — | — |
| Pericardial effusion | Cardiac disorders | — | — |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | — | — |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | — | — |
| Reaction | System | Placebo | TEZ/IVA |
|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — |
| Headache | Nervous system disorders | — | — |
| Nasopharyngitis | Infections and infestations | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | — | — |
| Abdominal pain upper | Gastrointestinal disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Fatigue | General disorders | — | — |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | — | — |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Bacterial test positive | Investigations | — | — |
| Decreased appetite | Metabolism and nutrition disorders | — | — |
Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Sepsis, Lower respiratory tract infection, Constipation, Multiple organ dysfunction syndrome, Suicidal ideation, Pericardial effusion, Musculoskeletal chest pain.
Data from ClinicalTrials.gov NCT03150719 adverse events section.
Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the cystic fibrosis transmembrane conductance regulator gene (CFTR) gene and who discontinued treatment with Orkambi due to respiratory symptoms considered related to treatment. This study is designed to evaluate the safety and efficacy of Tezacaftor/Ivacaftor (TEZ/IVA).
5 peer-reviewed publications reference this trial (live from Europe PMC):
Verify or expand the search:
Currently open trials in the same condition.
Trials by the same sponsor.
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03150719.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing