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Vertex Pharmaceuticals Incorporated clinical trials

Also registered as Vertex Pharmaceuticals
Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, Ireland, China and EU (CTIS) registers with Vertex Pharmaceuticals Incorporated as lead sponsor. Synced 26 Sep 2026
29Current trials
13Recruiting now
9Drugs in these trials
5Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Vertex Pharmaceuticals Incorporated is the lead sponsor of 29 current clinical trials in our USA, UK, Ireland, China and EU (CTIS) registers. 13 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are CTX001, Suzetrigine and Povetacicept. The most-studied condition is Cystic Fibrosis.

Company profile on this site (news, sites, medicines, approvals): Vertex Pharmaceuticals (US) →

ClinicalTrials.gov trials by status

  • Recruiting1348%
  • Active, closed1141%
  • By invitation311%

Trials by phase

  • Phase 31348%
  • Phase 2415%
  • Phase 1/2311%
  • Phase 1311%
  • Phase 2/327%
  • Observational14%
  • Phase 414%
01

Clinical trials sponsored by Vertex Pharmaceuticals Incorporated

27 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis
NCT07501702 · Myasthenia Gravis, Generalized
Phase 2 Recruiting 2026-05 UK, USA
Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN)
NCT07204275 · ClinicalTrials.gov ↗ · Primary Membranous Nephropathy
Phase 2/3 Recruiting 2025-09 Ireland, UK, USA, China
A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes
NCT04786262 · Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia
Phase 3 Recruiting 2021-03 UK, USA
Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease
NCT05312879 · ClinicalTrials.gov ↗ · Proteinuric Kidney Disease
Phase 2/3 Recruiting 2022-03 Ireland, UK, USA
Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral Neuropathy
NCT06628908 · Diabetic Peripheral Neuropathic Pain
Phase 3 Recruiting 2024-10 USA
Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral Neuropathy
NCT07231419 · Diabetic Peripheral Neuropathic Pain
Phase 3 Recruiting 2025-11 USA
A Phase 1, First-in-human Study of VX-433
NCT07584434 · ClinicalTrials.gov ↗ · Narcolepsy Type 1 (NT1)
Phase 1 Recruiting 2026-05 USA
Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy
NCT06619860 · ClinicalTrials.gov ↗ · Diabetic Peripheral Neuropathic Pain
Phase 2 Recruiting 2024-10 UK, USA
An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney Transplant
NCT06832410 · ClinicalTrials.gov ↗ · Type 1 Diabetes, Kidney Transplant
Phase 3 Recruiting 2025-03 USA
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants
NCT07437105 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 1 Recruiting 2026-02 USA
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants
NCT07283770 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 1 Recruiting 2025-12 USA
Evaluation of Pain Treatment After Total Knee Arthroplasty
NCT07538570 · ClinicalTrials.gov ↗ · Pain
Phase 4 Recruiting 2026-04 USA
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
NCT05477563 · ClinicalTrials.gov ↗ · Beta-Thalassemia, Thalassemia, Hematologic Diseases
Drug: CTX001
Phase 3 Recruiting 2022-08 USA
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
NCT05844449 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 3 By invitation 2023-08 UK, USA
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529 · ClinicalTrials.gov ↗ · Beta-Thalassemia, Thalassemia, Sickle Cell Disease
Drug: CTX001
Phase 3 By invitation 2021-01 UK, USA
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
NCT06926621 · ClinicalTrials.gov ↗ · Myotonic Dystrophy Type 1 (DM1)
Drug: VX-670
Phase 2 By invitation 2025-04 UK, USA
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
NCT05444257 · Cystic Fibrosis
Phase 3 Active, closed 2022-11 Ireland, UK, USA
A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
NCT05791201 · Type 1 Diabetes
Phase 1/2 Active, closed 2023-05 UK, USA
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
NCT06185764 · ClinicalTrials.gov ↗ · Myotonic Dystrophy Type 1 (DM1)
Drug: VX-670
Phase 1/2 Active, closed 2024-02 UK, USA
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
NCT05356195 · ClinicalTrials.gov ↗ · Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn
Drug: CTX001
Phase 3 Active, closed 2022-05 UK, USA
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05329649 · ClinicalTrials.gov ↗ · Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance
Drug: CTX001
Phase 3 Active, closed 2022-05 UK, USA
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
NCT06747572 · ClinicalTrials.gov ↗ · Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Observational Active, closed 2024-12 UK, USA
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
NCT05422222 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 3 Active, closed 2022-06 UK, USA
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
NCT06460506 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 3 Active, closed 2024-11 UK
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741 · ClinicalTrials.gov ↗ · Cystic Fibrosis
Phase 1/2 Active, closed 2023-02 UK, USA
Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)
NCT07161037 · ClinicalTrials.gov ↗ · Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Phase 2 Active, closed 2025-11 UK, USA
Evaluation of the Long-term Safety and Effectiveness of Suzetrigine (SUZ) in Participants With Painful Diabetic Peripheral Neuropathy (DPN)
NCT06696443 · ClinicalTrials.gov ↗ · Diabetic Peripheral Neuropathic Pain
Phase 3 Active, closed 2024-12 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Vertex Pharmaceuticals Incorporated in the EU trials register (CTIS)

2 trials · 1 in Ireland

Authorised or ongoing trials in the EU Clinical Trials Information System with Vertex Pharmaceuticals Incorporated as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX‑828/Deutivacaftor With and Without Tezacaftor in Subjects Aged 18 Years and Older With Cystic Fibrosis
2025-523400-72-00 · CTIS ↗ · Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Phase 2 Authorised Ireland + 10 more
A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects with Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants
2024-517393-13-00 · CTIS ↗ · Genetic Phenomena
Phase 2 Ongoing France, Germany, Italy + 3 more
03

Drugs in Vertex Pharmaceuticals Incorporated trials

9 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Vertex Pharmaceuticals Incorporated trials

22 conditions
Cystic Fibrosis7Diabetic Peripheral Neuropathic Pain4Beta-Thalassemia3Thalassemia3Hematologic Diseases3Type 1 Diabetes2Myotonic Dystrophy Type 1 (DM1)2Genetic Diseases, Inborn2Sickle Cell Disease2Autosomal Dominant Polycystic Kidney Disease (ADPKD)2Primary Membranous Nephropathy1Proteinuric Kidney Disease1Myasthenia Gravis, Generalized1Diabetes Mellitus, Type 11Impaired Hypoglycemic Awareness1Severe Hypoglycemia1Hydroxyurea Failure1Hydroxyurea Intolerance1Hemoglobinopathies1Narcolepsy Type 1 (NT1)1Kidney Transplant1Pain1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA2613
Clinical trials in UK185
Clinical trials in Ireland32
Clinical trials in China11
EU register (CTIS)2—

A trial with sites in more than one country appears in each of those registers.

06

Questions about Vertex Pharmaceuticals Incorporated trials

How many clinical trials does Vertex Pharmaceuticals Incorporated sponsor?

Vertex Pharmaceuticals Incorporated is the lead sponsor of 27 current trials on ClinicalTrials.gov in our USA, UK, Ireland and China registers, and 2 authorised or ongoing trials in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Vertex Pharmaceuticals Incorporated trials recruiting?

Yes. 13 of its 27 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Vertex Pharmaceuticals Incorporated testing?

Its current trials name 9 drugs and biologicals as interventions. The most frequent are CTX001 (4), Suzetrigine (4), Povetacicept (2), Pregabalin (2) and VX-121/TEZ/D-IVA (2). The number in brackets is how many of its trials name each drug.

Does Vertex Pharmaceuticals Incorporated have clinical trials in Ireland?

Yes. 3 trials in our Ireland register (ClinicalTrials.gov), and 1 trial in the EU register (CTIS) with a site in Ireland. Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.