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A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Phase 2 trial testing VX-561 in Cystic Fibrosis in 77 participants. Completed in 20 August 2020.
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 77 |
| Start date | 17 April 2019 |
| Primary completion | 20 August 2020 |
| Estimated completion | 20 August 2020 |
| Sites | 48 locations across Netherlands, Belgium, Ireland, United Kingdom, Germany, Australia, United States |
Vertex Pharmaceuticals Incorporated — full company profile →
18 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.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | -0.8 | -6.2 – 4.7 |
| VX-561: 150 mg | 3.1 | -0.8 – 7.0 |
| VX-561: 250 mg | 2.7 | -1.0 – 6.5 |
Sweat samples were collected using an approved collection device.
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 0.9 | -9.5 – 11.3 |
| VX-561: 150 mg | 3.3 | -4.6 – 11.2 |
| VX-561: 250 mg | -6.5 | -14.1 – 1.2 |
| Group | Value | 95% CI |
|---|---|---|
| VX-561: 25 mg | 26.1 | ± 24.7 |
| VX-561: 50 mg | 123 | ± 61.6 |
| VX-561: 150 mg | 458 | ± 273 |
| VX-561: 250 mg | 1100 | ± 856 |
| Group | Value | 95% CI |
|---|---|---|
| VX-561: 25 mg | 18.1 | ± 17.7 |
| VX-561: 50 mg | 108 | ± 58.6 |
| VX-561: 150 mg | 378 | ± 213 |
| VX-561: 250 mg | 739 | ± 407 |
| Group | Value | 95% CI |
|---|---|---|
| VX-561: 25 mg | NA | ± NA |
| VX-561: 50 mg | 59.8 | ± 34.0 |
| VX-561: 150 mg | 211 | ± 189 |
| VX-561: 250 mg | 370 | ± 233 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 952 | ± 766 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 1330 | ± 774 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 662 | ± 398 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 8 | |
| VX-561: 25 mg | 4 | |
| VX-561: 50 mg | 8 | |
| VX-561: 150 mg | 21 | |
| VX-561: 250 mg | 23 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor | 1 | |
| VX-561: 25 mg | 2 | |
| VX-561: 50 mg | 2 | |
| VX-561: 150 mg | 2 | |
| VX-561: 250 mg | 1 |
Time frame: Baseline up to Week 16. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Ivacaftor | VX-561: 25 mg | VX-561: 50 mg | VX-561: 150 mg | VX-561: 250 mg |
|---|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — | — | — | — |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | — | — | — | — | — |
| Forced expiratory volume decreased | Investigations | — | — | — | — | — |
| Reaction | System | Ivacaftor | VX-561: 25 mg | VX-561: 50 mg | VX-561: 150 mg | VX-561: 250 mg |
|---|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — | — | — | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Nasopharyngitis | Infections and infestations | — | — | — | — | — |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Abdominal distension | Gastrointestinal disorders | — | — | — | — | — |
| Abdominal pain upper | Gastrointestinal disorders | — | — | — | — | — |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | — | — | — | — | — |
| Sinusitis | Infections and infestations | — | — | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — | — | — |
| Aspartate aminotransferase increased | Investigations | — | — | — | — | — |
| Blood creatine phosphokinase increased | Investigations | — | — | — | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — | — | — | — |
| Migraine | Nervous system disorders | — | — | — | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | — | — | — | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — | — | — | — |
| Leukocytosis | Blood and lymphatic system disorders | — | — | — | — | — |
| Palpitations | Cardiac disorders | — | — | — | — | — |
| Ear pain | Ear and labyrinth disorders | — | — | — | — | — |
| Tinnitus | Ear and labyrinth disorders | — | — | — | — | — |
| Glaucoma | Eye disorders | — | — | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — | — | — |
| Abdominal pain lower | Gastrointestinal disorders | — | — | — | — | — |
| Constipation | Gastrointestinal disorders | — | — | — | — | — |
| Duodenitis | Gastrointestinal disorders | — | — | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — | — | — |
| Vomiting | Gastrointestinal disorders | — | — | — | — | — |
| Chest discomfort | General disorders | — | — | — | — | — |
| Fatigue | General disorders | — | — | — | — | — |
| Malaise | General disorders | — | — | — | — | — |
| Pyrexia | General disorders | — | — | — | — | — |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | — | — | — | — | — |
| Epididymitis | Infections and infestations | — | — | — | — | — |
| Fungal skin infection | Infections and infestations | — | — | — | — | — |
| Oral candidiasis | Infections and infestations | — | — | — | — | — |
Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Distal intestinal obstruction syndrome, Forced expiratory volume decreased.
Data from ClinicalTrials.gov NCT03911713 adverse events section.
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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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/NCT03911713.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing