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Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Phase 2 trial testing VX-814 in Alpha 1-Antitrypsin Deficiency in 48 participants. Terminated before completion.
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Phase | Phase 2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 48 |
| Start date | 13 January 2020 |
| Primary completion | 14 November 2020 |
| Estimated completion | 14 November 2020 |
| Sites | 28 locations across Ireland, Canada, United States, Germany |
Vertex Pharmaceuticals Incorporated — full company profile →
Adults 18 to 80, any sex, with Alpha 1-Antitrypsin Deficiency. 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 |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | -0.4 | ± 0.3 |
| Part A1: VX-814 100 mg | 0.2 | ± 0.1 |
| Part A1: VX-814 200 mg | 0.3 | ± 0.5 |
| Parts A1 and A2 Combined: VX-814 400 mg | 1.4 | ± 0.6 |
| Part B: VX-814 600 mg | 1.6 | ± 1.0 |
| Group | Value | 95% CI |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | 4 | |
| Part A1: VX-814 100 mg | 3 | |
| Part A1: VX-814 200 mg | 1 | |
| Parts A1 and A2 Combined: VX-814 400 mg | 10 | |
| Part B: VX-814 600 mg | 14 |
| Group | Value | 95% CI |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | 0 | |
| Part A1: VX-814 100 mg | 2 | |
| Part A1: VX-814 200 mg | 1 | |
| Parts A1 and A2 Combined: VX-814 400 mg | 1 | |
| Part B: VX-814 600 mg | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | 0.4 | ± 1.3 |
| Part A1: VX-814 100 mg | 0.1 | ± 0.2 |
| Part A1: VX-814 200 mg | 0.7 | ± 0.7 |
| Parts A1 and A2 Combined: VX-814 400 mg | 2.4 | ± 1.2 |
| Part B: VX-814 600 mg | 2.6 | ± 1.1 |
| Group | Value | 95% CI |
|---|---|---|
| Part A1: VX-814 100 mg | 0.476 | ± 0.375 |
| Part A1: VX-814 200 mg | 0.948 | ± 0.512 |
| Parts A1 and A2 Combined: VX-814 400 mg | 3.53 | ± 1.72 |
| Part B: VX-814 600 mg | 10.3 | ± 6.55 |
| Group | Value | 95% CI |
|---|---|---|
| Part A1: VX-814 100 mg | 0.244 | ± 0.0919 |
| Part A1: VX-814 200 mg | 1.07 | ± 0.152 |
| Parts A1 and A2 Combined: VX-814 400 mg | 3.25 | ± 2.23 |
| Part B: VX-814 600 mg | 8.53 | ± 11.8 |
| Group | Value | 95% CI |
|---|---|---|
| Part A1: VX-814 100 mg | 0.700 | |
| Part A1: VX-814 200 mg | 1.45 | |
| Parts A1 and A2 Combined: VX-814 400 mg | 3.68 | ± 3.40 |
| Part B: VX-814 600 mg | 7.20 | ± 6.05 |
| Group | Value | 95% CI |
|---|---|---|
| Part A1: VX-814 100 mg | 0.326 | ± 0.0282 |
| Part A1: VX-814 200 mg | 0.993 | ± 0.0300 |
| Parts A1 and A2 Combined: VX-814 400 mg | 3.23 | ± 2.82 |
| Part B: VX-814 600 mg | 5.80 | ± 3.73 |
Time frame: Day 1 up to Week 8. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Parts A1, A2 and B Combine… | Part A1: VX-814 100 mg | Part A1: VX-814 200 mg | Parts A1 and A2 Combined: … | Part B: VX-814 600 mg |
|---|---|---|---|---|---|---|
| Gastrointestinal infection | Infections and infestations | — | — | — | — | — |
| Alanine aminotransferase increased | Investigations | — | — | — | — | — |
| Aspartate aminotransferase increased | Investigations | — | — | — | — | — |
| Rash erythematous | Skin and subcutaneous tissue disorders | — | — | — | — | — |
| Reaction | System | Parts A1, A2 and B Combine… | Part A1: VX-814 100 mg | Part A1: VX-814 200 mg | Parts A1 and A2 Combined: … | Part B: VX-814 600 mg |
|---|---|---|---|---|---|---|
| Alanine aminotransferase increased | Investigations | — | — | — | — | — |
| Aspartate aminotransferase increased | Investigations | — | — | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — | — | — |
| Fatigue | General disorders | — | — | — | — | — |
| Urinary tract infection | Infections and infestations | — | — | — | — | — |
| Blood pressure increased | Investigations | — | — | — | — | — |
| Headache | Nervous system disorders | — | — | — | — | — |
| Palpitations | Cardiac disorders | — | — | — | — | — |
| Abdominal distension | Gastrointestinal disorders | — | — | — | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — | — | — | — |
| Frequent bowel movements | Gastrointestinal disorders | — | — | — | — | — |
| Vomiting | Gastrointestinal disorders | — | — | — | — | — |
| Ear infection | Infections and infestations | — | — | — | — | — |
| Respiratory tract infection | Infections and infestations | — | — | — | — | — |
| Rhinitis | Infections and infestations | — | — | — | — | — |
| Tooth abscess | Infections and infestations | — | — | — | — | — |
| Vulvovaginal candidiasis | Infections and infestations | — | — | — | — | — |
| Dental restoration failure | Injury, poisoning and procedural complications | — | — | — | — | — |
| Blood cholesterol increased | Investigations | — | — | — | — | — |
| Blood creatine phosphokinase increased | Investigations | — | — | — | — | — |
| Crystal urine present | Investigations | — | — | — | — | — |
| Eosinophil count increased | Investigations | — | — | — | — | — |
| Gamma-glutamyltransferase increased | Investigations | — | — | — | — | — |
| International normalised ratio increased | Investigations | — | — | — | — | — |
| Protein urine present | Investigations | — | — | — | — | — |
| Prothrombin time prolonged | Investigations | — | — | — | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — | — | — | — |
| Bone pain | Musculoskeletal and connective tissue disorders | — | — | — | — | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — | — | — | — | — |
| Restless legs syndrome | Nervous system disorders | — | — | — | — | — |
| Nightmare | Psychiatric disorders | — | — | — | — | — |
| Pollakiuria | Renal and urinary disorders | — | — | — | — | — |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | — | — | — | — | — |
Most-reported serious reactions: Gastrointestinal infection, Alanine aminotransferase increased, Aspartate aminotransferase increased, Rash erythematous.
Data from ClinicalTrials.gov NCT04167345 adverse events section.
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
3 peer-reviewed publications reference this trial (live from Europe PMC):
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