Clinical trials: sponsored by Vertex Pharmaceuticals Incorporated
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)
The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.
- Trial name
- AGLOW (NCT07161037)
- Testing
- VX-407
- Where
- 43 sites in United States, Belgium and 6 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pain
Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants
The purpose of the study is to evaluate the pharmacokinetics (PK) of suzetrigine (SUZ) and its active metabolite in breast milk of lactating female participants.
- Trial name
- NCT07378865
- Testing
- Suzetrigine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness
A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes
This study will evaluate the safety, tolerability and efficacy of VX-880 infusion and VX-017 infusion in participants with type 1 diabetes (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.
- Conditions
- Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia
- Trial name
- NCT04786262
- Testing
- VX-880, VX-017
- Where
- 29 sites in United States, Canada and 8 more
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Proteinuric Kidney Disease
Inaxaplin in Participants With Proteinuric APOL1 Mediated Kidney Disease With or Without Comorbidities
The purpose of the study is to evaluate the efficacy, safety, and tolerability of Inaxaplin (IXP) in participants with proteinuric APOL1- mediated kidney disease (AMKD).
- Trial name
- AMPLIFIED (NCT06794996)
- Testing
- Inaxaplin
- Where
- 35 sites in United States
- Volunteers
- 42 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pain
Effects of Efavirenz on the Pharmacokinetics of Suzetrigine in Healthy Participants
The purpose of the study is to evaluate the pharmacokinetics (PK) of Suzetrigine (SUZ) and its metabolite in the absence and presence of efavirenz, a moderate CYP3A inducer.
- Trial name
- NCT07570069
- Testing
- Suzetrigine, Efavirenz
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX-118 or TEZ/ deutivacaftor (D-IVA) in healthy participants and VX-828 in…
- Trial name
- NCT06154447
- Testing
- VX-828, Itraconazole, Midazolam
- Where
- 12 sites in United States
- Volunteers
- 165 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Diabetic Peripheral Neuropathic Pain
Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral Neuropathy
The purpose of this study is to evaluate the efficacy, safety, and tolerability of Suzetrigine in participants with pain associated with diabetic peripheral neuropathy (DPN).
- Trial name
- NCT07231419
- Testing
- Suzetrigine
- Where
- 75 sites in United States
- Volunteers
- 734 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Hydroxyurea Failure
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human…
- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
- Trial name
- NCT05329649
- Testing
- CTX001
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 13 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Narcolepsy Type 1 (NT1)
A Phase 1, First-in-human Study of VX-433
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-433 following single and multiple ascending doses, as well as to assess the effect of VX-433 on the PK of midazolam, bupropion…
- Trial name
- NCT07584434
- Testing
- VX-433, Midazolam, Bupropion
- Where
- United States
- Volunteers
- 118 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).
- Trial name
- NCT06460506
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 18 sites in Australia, Canada and 5 more
- Volunteers
- 50 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Diabetic Peripheral Neuropathic Pain
Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy
The purpose of this study is to evaluate the efficacy, safety, and tolerability of VX-993 in participants with pain associated with Diabetic Peripheral Neuropathy (DPN)
- Trial name
- NCT06619860
- Testing
- VX-993, Pregabalin
- Where
- 47 sites in United States, Canada and 4 more
- Volunteers
- 300 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Acute Pain
A Study Evaluating Efficacy and Safety of VX-993 for Acute Pain After a Bunionectomy
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of VX-993 in treating acute pain after a bunionectomy.
- Trial name
- NCT06619847
- Testing
- VX-993, HB/APAP
- Where
- 14 sites in United States
- Volunteers
- 367 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR)…
- Trial name
- NCT05422222
- Testing
- VX-121/TEZ/D-IVA
- Where
- 38 sites in United States, Australia and 8 more
- Volunteers
- 210 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT05844449
- Testing
- VNZ/TEZ/D-IVA
- Where
- 37 sites in United States, Australia and 8 more
- Volunteers
- 174 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants
The purpose of this study is to evaluate the effect of multiple doses of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) on the PK of rosuvastatin and the safety and tolerability of coadministration of VNZ/TEZ/D-IVA with…
- Trial name
- NCT07349394
- Testing
- VNZ/TEZ/D-IVA, Rosuvastatin
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants
The purpose of the study is to evaluate the effect of VX-407 on the pharmacokinetics of levonorgestrel (LNG) and ethinyl estradiol (EE), norgestimate (NGM) and EE, norethindrone (NET) and EE and drospirenone (DRSP) and EE.
- Trial name
- NCT07074327
- Testing
- VX-407, LNG/EE, NGM/EE
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated