224 completed, 21 terminated, 15 recruiting, 12 active not recruiting. 96 Phase 3, 92 Phase 1, 88 Phase 2, 10 Phase 4, 5 Not applicable.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype Exa-cel - Sickle Cell Disease | Phase 3 | Withdrawn | Vertex Pharmaceuticals Incorporated | 2027-07-31 | 2026-03-25 | |
| Suzetrigine Added to Multimodal Analgesia for Women Undergoing Minimally Invasive Hysterectomy Suzetrigine - Postoperative Pain | Phase 4 | Not yet recruiting | Duke University | 123 | 2026-11-01 | 2026-09-23 |
| A Phase 3b Study of Participant Experience on Povetacicept Administered Via Autoinjector as Compared to Sibeprenlimab Administered Via Prefilled Syringe Povetacicept, Sibeprenlimab - Immunoglobulin A Nephropathy | Phase 3 | Not yet recruiting | Vertex Pharmaceuticals Incorporated | 40 | 2026-10-23 | 2026-10-22 |
| Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose 14C-VX-407 - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Not yet recruiting | Vertex Pharmaceuticals Incorporated | 8 | 2026-10-19 | 2026-10-09 |
| Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension DM1, Myotonic Dystrophy | Recruiting | Virginia Commonwealth University | 1000 | 2026-10-15 | 2026-10-16 | |
| Assessing Suzetrigine for Pain Reduction Following Ureteroscopic Surgery multimodal analgesia regimen without suzetrigine, Suzetrigine - Opioid Consumption, Postoperative, Multimodal Analgesia | Phase 3 | Not yet recruiting | Brigham and Women's Hospital | 100 | 2026-10-01 | 2026-09-18 |
| Suzetrigine for Postoperative Pain After Total Ankle Replacement Suzetrigine - Ankle Osteoarthritis, Postoperative Pain | Phase 4 | Not yet recruiting | Soheil Ashkani Esfahani | 28 | 2026-10-01 | 2026-09-30 |
| A Phase 1 Relative Bioavailability Study of a VX-581 Tablet Formulation VX-581 - Cystic Fibrosis | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 16 | 2026-09-23 | 2026-10-19 |
| A Phase 3 Open-Label Study of Long-term Treatment With Povetacicept in Adults With Immunoglobulin A Nephropathy Povetacicept - Immunoglobulin A Nephropathy | Phase 3 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 605 | 2026-09-17 | 2026-10-19 |
| Effects of VX-407 on the Pharmacokinetics of Combined Oral Contraceptives VX-407, LNG/EE, NGM/EE - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 148 | 2026-09-01 | 2026-09-30 |
| Role of Suzetrigine as a Part of a Multimodal Regimen to Reduce Pain and Opioid Use After Total Knee Arthroplasty Suzetrigine - Knee Arthritis, Knee Osteoarthritis | Phase 4 | Not yet recruiting | Emory University | 75 | 2026-07-01 | 2026-06-03 |
| A Phase 1, First-in-human Study of VX-433 VX-433, Placebo, Midazolam - Narcolepsy Type 1 (NT1) | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 118 | 2026-05-21 | 2026-06-08 |
| A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis Povetacicept, Placebo - Myasthenia Gravis, Generalized | Phase 2 | Recruiting | Vertex Pharmaceuticals Incorporated | 30 | 2026-05-13 | 2026-09-11 |
| Effects of Efavirenz on the Pharmacokinetics of Suzetrigine in Healthy Participants Suzetrigine, Efavirenz - Pain | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 18 | 2026-05-07 | 2026-08-18 |
| Evaluation of Pain Treatment After Total Knee Arthroplasty Suzetrigine, Placebo - Pain | Phase 4 | Recruiting | Vertex Pharmaceuticals Incorporated | 60 | 2026-04-24 | 2026-08-18 |
| Suzetrigine for Non-Mastectomy Breast Surgery suzetrigine - Pain, Acute Postoperative, Breast Disease | Phase 4 | Enrolling by invitation | University of California, San Diego | 120 | 2026-04-14 | 2026-09-16 |
| Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants VX-272, Placebo, Tezacaftor - Cystic Fibrosis | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 160 | 2026-02-25 | 2026-09-23 |
| Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants Suzetrigine - Pain | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 13 | 2026-02-13 | 2026-09-09 |
| Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants VNZ/TEZ/D-IVA, Rosuvastatin - Cystic Fibrosis | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 18 | 2026-01-12 | 2026-04-02 |
| Evaluation of VX-581 in Healthy Participants and in Participants With Cystic Fibrosis VX-581, Placebo, D-IVA - Cystic Fibrosis | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 152 | 2025-12-09 | 2026-10-19 |
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Cystic Fibrosis (131) · Hepatitis C (30) · Pain (26) · Hepatitis C, Chronic (21) · Acute Pain (11) · Genetic Diseases, Inborn (11) · Polycystic Kidney, Autosomal Dominant (7) · Arthritis, Rheumatoid (6) · Hematologic Diseases (6) · Hemoglobinopathies (6) · Anemia, Sickle Cell (5) · Infant, Newborn, Diseases (5) · Lung Diseases (5) · Pancreatic Diseases (5) · alpha 1-Antitrypsin Deficiency (5)
ivacaftor (98) · telaprevir (37) · Ribavirin (30) · elexacaftor, ivacaftor, tezacaftor drug combination (24) · peginterferon alfa-2a (21) · tezacaftor (18) · tezacaftor, ivacaftor drug combination (18) · lumacaftor (15) · lumacaftor, ivacaftor drug combination (10) · deutivacaftor, tezacaftor , vanzacaftor (8) · Midazolam (7) · exagamglogene autotemcel (7) · 2-((2-(1H-pyrrolo(2,3-b)pyridin-3-yl)pyrimidin-4-yl)amino)-2-methyl-N-(2,2,2-trifluoroethyl)butanamide (6) · lomibuvir (5) · Itraconazole (4)
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Fumitaka Suzuki, MD (6) · Robert Kauffman, MD, PhD (4) · H. Frank Farmer, MD (3) · Virginia Stallings, MD (3) · Bradley Bloom, MD, FACR, FAAP (2) · Karl Donn (2) · Patrick A Flume, MD (2) · Adebayo Lawal, M.D. (1) · Ajay Premkumar, MD (1) · Alan Smyth (1) · Alan Smyth, Prof (1) · Ali Ashrafzadeh, MD, FACR (1)
PMID 37983082, PMID 33331662, PMID 36509366, PMID 19404957, PMID 22239511, PMID 24982088, PMID 25981758, PMID 26070913, PMID 27298017, PMID 30146268, PMID 30334693, PMID 30587335, PMID 33283989, PMID 34175041, PMID 36842446, PMID 37530822, PMID 39756424, PMID 40857358, PMID 40862696, PMID 41384808, PMID 41689837, PMID 42252696, PMID 42274009, PMID 17285598, PMID 19403902, PMID 19403903, PMID 19852040, PMID 20375406, PMID 21083385, PMID 21381984, PMID 21690488, PMID 21696307, PMID 21825083, PMID 21827730, PMID 21916639, PMID 22239508, PMID 22383668, PMID 23190247, PMID 23383655, PMID 23478952, PMID 23590265, PMID 23685940, PMID 23844986, PMID 24316262, PMID 24461666, PMID 24660233, PMID 24811404, PMID 24901821, PMID 24973281, PMID 25311995
2017 (12), 2018 (9), 2016 (8), 2019 (7), 2023 (5), 2014 (4), 2015 (4), 2009 (3), 2025 (3), P30DK072482 (1), R34HL109334 (1), R34HL127166 (1)