A Phase 3 interventional study of ELX/TEZ/IVA and IVA in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 29 sites in 4 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2023-07-28.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
1,605 studies on the registry are indexed under Cystic Fibrosis; 196 are open to participants now.
This study's enrollment of 172 is above the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Vertex Pharmaceuticals Incorporated is the lead sponsor of 249 studies on the registry; 20 are open to participants now.
Of its 106 completed or terminated interventional studies of FDA-regulated products, 53 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
Drug: ELX/TEZ/IVA · Drug: IVA
Drug pages: Elexacaftor · Tezacaftor · Ivacaftor
Fixed-dose combination (FDC) tablet for oral administration.
Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Tablet for oral administration.
Also known as: VX-770, ivacaftor
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 52
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 86
| Milestone | ELX/TEZ/IVA |
|---|---|
| Started | 172 |
| Completed | 159 |
| Not completed | 13 |
| Withdrew: Adverse event | 2 |
| Withdrew: Withdrawal of consent (not due to ae) | 4 |
| Withdrew: Commercial drug is available for participant | 2 |
| Withdrew: Other | 5 |
| Milestone | ELX/TEZ/IVA |
|---|---|
| Started | 50 |
| Completed | 0 |
| Not completed | 50 |
| Withdrew: Commercial drug is available for participant | 46 |
| Withdrew: Other | 4 |
| Participants | Part A: ELX/TEZ/IVA |
|---|---|
| Participants with TEAEs | 160 |
| Participants with SAEs | 26 |
| Participants | Part B: ELX/TEZ/IVA |
|---|---|
| Participants with TEAEs | 50 |
| Participants with SAEs | 8 |
Collected over Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: ELX/TEZ/IVA | 0/172 (0%) | 26/172 (15.1%) | 127/172 (73.8%) |
| Part B: ELX/TEZ/IVA | 0/50 (0%) | 8/50 (16%) | 47/50 (94%) |
| Event | Part A: ELX/TEZ/IVA | Part B: ELX/TEZ/IVA |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 8/172 | 5/50 |
| CholecystitisHepatobiliary disorders | 0/172 | 1/50 |
| Limb traumatic amputationInjury, poisoning and procedural complications | 0/172 | 1/50 |
| Forced expiratory volume decreasedInvestigations | 0/172 | 1/50 |
| Productive coughRespiratory, thoracic and mediastinal disorders | 0/172 | 1/50 |
| Alanine aminotransferase increasedInvestigations | 2/172 | 0/50 |
| Aspartate aminotransferase increasedInvestigations | 2/172 | 0/50 |
| Atrial fibrillationCardiac disorders | 1/172 | 0/50 |
| Myocardial infarctionCardiac disorders | 1/172 | 0/50 |
| Sinus arrestCardiac disorders | 1/172 | 0/50 |
| Event | Part A: ELX/TEZ/IVA | Part B: ELX/TEZ/IVA |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 17/172 | 21/50 |
| COVID-19Infections and infestations | 2/172 | 17/50 |
| NasopharyngitisInfections and infestations | 28/172 | 16/50 |
| HeadacheNervous system disorders | 42/172 | 10/50 |
| Upper respiratory tract infectionInfections and infestations | 10/172 | 11/50 |
| PyrexiaGeneral disorders | 7/172 | 8/50 |
| Immunisation reactionImmune system disorders | 0/172 | 7/50 |
| Vaccination complicationInjury, poisoning and procedural complications | 24/172 | 0/50 |
| CoughRespiratory, thoracic and mediastinal disorders | 23/172 | 5/50 |
| Productive coughRespiratory, thoracic and mediastinal disorders | 4/172 | 6/50 |
Baseline data is based on the parent study baseline, which is defined as the most recent non-missing measurements collected before the first dose of the study drug in the treatment period of parent studies. Baseline data is presented for participants who received at least 1 dose of study drug in this study.
| Age, Continuous(years) | ELX/TEZ/IVA |
|---|---|
| Part A | 27.9 ± 11.4 |
| Part B | 25.6 ± 12.0 |
| Sex: Female, Male(Participants) | ELX/TEZ/IVA |
|---|---|
| Part A — Female | 87 |
| Part A — Male | 85 |
| Part B — Female | 26 |
| Part B — Male | 24 |
| Race/Ethnicity, Customized(Participants) | ELX/TEZ/IVA |
|---|---|
| Part A — Hispanic or Latino | 3 |
| Part A — Not Hispanic or Latino | 167 |
| Part A — Not collected per local regulations | 2 |
| Part B — Hispanic or Latino | 1 |
| Part B — Not Hispanic or Latino | 49 |
| Part B — Not collected per local regulations | 0 |
| Race/Ethnicity, Customized(Participants) | ELX/TEZ/IVA |
|---|---|
| Part A — White | 169 |
| Part A — Black or African American | 0 |
| Part A — Asian | 2 |
| Part A — American Indian or Alaska Native | 0 |
| Part A — Native Hawaiian or Other Pacific Islander | 0 |
| Part A — White, Asian | 1 |
| Part A — Not collected per local regulations | 0 |
| Part B — White | 50 |
| Part B — Black or African American | 0 |
| Part B — Asian | 0 |
| Part B — American Indian or Alaska Native | 0 |
| Part B — Native Hawaiian or Other Pacific Islander | 0 |
| Part B — White, Asian | 0 |
| Part B — Not collected per local regulations | 0 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No — Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.
This study is completed, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Elexacaftor · Tezacaftor · Ivacaftor
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