Clinical trials: sponsored by Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator…
- Trial name
- NCT05331183
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 81 sites in Austria, Belgium and 13 more
- Volunteers
- 297 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.
- Trial name
- NCT06747572
- Where
- 45 sites in United States, Belgium and 7 more
- Volunteers
- 401 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Proteinuric Kidney Disease
Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.
- Trial name
- AMPLITUDE (NCT05312879)
- Testing
- VX-147
- Where
- 314 sites in United States, Belgium and 11 more
- Volunteers
- 522 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age.
- Trial name
- NCT05882357
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Canada and 5 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
- Trial name
- NCT05444257
- Testing
- VX-121/TEZ/D-IVA
- Where
- 195 sites in United States, Australia and 21 more
- Volunteers
- 822 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
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
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
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
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
Sickle Cell Disease
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
- Trial name
- NCT05951205
- Testing
- Exa-cel
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.
- Trial name
- NCT05153317
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 22 sites in United States, Australia and 3 more
- Volunteers
- 71 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
- Trial name
- NCT06506773
- Where
- 50 sites in United States, Australia and 9 more
- Volunteers
- 79 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del…
- Trial name
- NCT03525574
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 110 sites in United States, Australia and 11 more
- Volunteers
- 507 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
- Trial name
- NCT04969224
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Belgium and 2 more
- Volunteers
- 82 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT04183790
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 21 sites in United States, Australia and 3 more
- Volunteers
- 64 people
- Sponsor
- Vertex Pharmaceuticals Incorporated