Vertex Pharmaceuticals Clinical Trials: 259 Led Studies

Archive historyOct 2022 to Jun 2026

146 recorded changes across Vertex Pharmaceuticals trials

Every status move, completion date shift and enrollment change ComplyRx has recorded across 40 of these trials. One is shown below.

Enrollment changeEnrollment finalised at 42NCT06794996 · recorded 1 Jun 2026

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+142 more recorded changes · Track a drug to see every change the night it lands

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Most active programmes

The drugs appearing most often across Vertex Pharmaceuticals's recently active trials. Comparator arms are excluded.

IVA14 trials
Suzetrigine13 trials
ELX/TEZ/IVA11 trials
VX-54811 trials
VX-9938 trials
CTX0016 trials

Upcoming readouts

Next primary completion dates among Vertex Pharmaceuticals's active phase 2 and phase 3 trials. Dates are registry estimates and move often, which is exactly what the tracker watches.

Sept 2026An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With …Phase 3
Mar 2027Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropa…Phase 2
Apr 2027Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Periphe…Phase 3
May 2027Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral Ne…Phase 3
Jun 2027Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-De…Phase 3
Jun 2027A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 DiabetesPhase 3

Top therapeutic areas

Conditions studied most often across Vertex Pharmaceuticals's recently active trials.

Cystic Fibrosis28 trials
Pain23 trials
Acute Pain7 trials
Hemoglobinopathies6 trials
Sickle Cell Disease5 trials
Autosomal Dominant Polycystic Kidney Disease (ADPKD)5 trials

Vertex Pharmaceuticals trials

The 50 most significant of Vertex Pharmaceuticals's recently active trials, soonest estimated readout first.
Select any row for the full record; the change timeline unlocks with tracking.

Trial	Phase	Stability	Status	Enrollment	Est. readout
An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney TransplantNCT06832410Biological/Vaccine	Phase 3	Locked · track to reveal	OngoingRecruiting	10 est.	17 Sept 2026
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination TherapyNCT05444257VX-121/TEZ/D-IVA	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	822 est.	30 Oct 2026
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy ParticipantsNCT07283770VX-581	Phase 1	Locked · track to reveal	OngoingRecruiting	136 est.	19 Nov 2026
AMPLIFIEDInaxaplin in Participants With Proteinuric APOL1 Mediated Kidney Disease With or Without ComorbiditiesNCT06794996Inaxaplin	Phase 2	Locked · track to reveal	OngoingActive, not recruiting	42 est.	30 Dec 2026
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney DiseaseNCT06747572	—	Not yet scored	OngoingActive, not recruiting	401 est.	31 Dec 2026
Evaluation of the Long-term Safety and Effectiveness of Suzetrigine (SUZ) in Participants With Painful Diabetic Peripheral Neuropathy (DPN)NCT06696443Suzetrigine	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	455 est.	25 Jan 2027
Evaluation of Pain Treatment After Total Knee ArthroplastyNCT07538570Suzetrigine	Phase 4	Locked · track to reveal	OngoingRecruiting	60 est.	30 Jan 2027
GalileoA Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)NCT06185764VX-670	Phase 1	Locked · track to reveal	OngoingRecruiting	52 est.	2 Feb 2027
Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female ParticipantsNCT07378865Suzetrigine	Phase 1	Locked · track to reveal	OngoingRecruiting	12 est.	26 Feb 2027
A Phase 1, First-in-human Study of VX-433NCT07584434VX-433	Phase 1	Locked · track to reveal	OngoingRecruiting	118 est.	5 Mar 2027
A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 DiabetesNCT05791201VX-264	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	7 est.	12 Mar 2027
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy ParticipantsNCT07437105VX-272	Phase 1	Locked · track to reveal	OngoingRecruiting	128 est.	23 Mar 2027
Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral NeuropathyNCT06619860VX-993	Phase 2	Locked · track to reveal	OngoingRecruiting	300 est.	25 Mar 2027
Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral NeuropathyNCT07231419Suzetrigine	Phase 3	Locked · track to reveal	OngoingRecruiting	734 est.	6 Apr 2027
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508delNCT05331183ELX/TEZ/IVA	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	297 est.	6 Apr 2027
Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral NeuropathyNCT06628908Suzetrigine	Phase 3	Locked · track to reveal	OngoingRecruiting	1,100 est.	31 May 2027
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)NCT05329649CTX001	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	13 est.	6 Jun 2027
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseNCT05477563CTX001	Phase 3	Locked · track to reveal	OngoingRecruiting	26 est.	9 Jun 2027
A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 DiabetesNCT04786262VX-880	Phase 3	Locked · track to reveal	OngoingRecruiting	52 est.	30 Jun 2027
AGLOWPhase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)NCT07161037VX-407	Phase 2	Locked · track to reveal	OngoingRecruiting	24 est.	22 Jul 2027
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and OlderNCT06460506ELX/TEZ/IVA	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	50 est.	30 Sept 2027
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)NCT05356195CTX001	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	16 est.	14 Nov 2027
Evaluation of Efficacy of Povetacicept in Adults With Immunoglobulin A Nephropathy (IgAN)NCT06564142Povetacicept	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	605 est.	30 Jan 2028
RUBY-3An Open-label Study of Povetacicept in Autoantibody-Associated Glomerular DiseasesNCT05732402Povetacicept	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	72 est.	2 Mar 2028
AMPLITUDEPhase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney DiseaseNCT05312879VX-147	Phase 2	Locked · track to reveal	OngoingRecruiting	466 est.	2 Jun 2028
OLYMPUSEvaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN)NCT07204275Povetacicept	Phase 2	Locked · track to reveal	OngoingRecruiting	176 est.	29 Dec 2028
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type INCT06926621VX-670	Phase 2	Locked · track to reveal	OngoingEnrolling by invitation	44 est.	30 Jan 2029
ETNAA Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia GravisNCT07501702Povetacicept	Phase 2	Locked · track to reveal	OngoingRecruiting	30 est.	2 Mar 2029
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and OlderNCT05844449VNZ/TEZ/D-IVA	Phase 3	Locked · track to reveal	OngoingEnrolling by invitation	174 est.	30 Jul 2029
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of AgeNCT05422222VX-121/TEZ/D-IVA	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	210 est.	30 Jun 2030
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC GenotypeNCT05951205Exa-cel	Phase 3	Locked · track to reveal	StoppedWithdrawn	0 est.	31 Dec 2033
A Long-term Follow-up Study in Participants Who Received CTX001NCT04208529CTX001	Phase 3	Locked · track to reveal	OngoingEnrolling by invitation	160 est.	30 Sept 2039
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic FibrosisNCT06154447VX-828	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	165 est.	16 Jul 2026
RUBY-4An Open-label Study of Povetacicept in Participants With Autoimmune CytopeniasNCT05757570povetacicept	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	30 est.	Jul 2026
Effects of Efavirenz on the Pharmacokinetics of Suzetrigine in Healthy ParticipantsNCT07570069Suzetrigine	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	18 est.	11 Jul 2026
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)NCT05668741VX-522 mRNA therapy	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	26 est.	21 Apr 2026
Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy ParticipantsNCT07349394VNZ/TEZ/D-IVA	Phase 1	Locked · track to reveal	Completed	18 est.	28 Feb 2026
A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy ParticipantsNCT07022119VX-407	Phase 1	Locked · track to reveal	Completed	32 est.	24 Feb 2026
Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy ParticipantsNCT07074327VX-407	Phase 1	Locked · track to reveal	Completed	74 est.	20 Feb 2026
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and OlderNCT05153317ELX/TEZ/IVA	Phase 3	Locked · track to reveal	Completed	71 est.	29 Jan 2026
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)NCT06506773	—	Not yet scored	Completed	79 est.	27 Nov 2025
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-ThalassemiaNCT03655678CTX001	Phase 2	Locked · track to reveal	Completed	59 est.	13 Nov 2025
A Single-arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Selected SurgeriesNCT06887959Suzetrigine	Phase 4	Locked · track to reveal	Completed	47 est.	27 Oct 2025
A Single-Arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Aesthetic or Reconstructive SurgeriesNCT06887972Suzetrigine	Phase 4	Locked · track to reveal	Completed	100 est.	24 Oct 2025
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of AgeNCT05882357ELX/TEZ/IVA	Phase 3	Locked · track to reveal	Completed	70 est.	4 Sept 2025
A Study to Evaluate the Relative Bioavailability of Povetacicept Formulations and Bioequivalence of Povetacicept PresentationsNCT07010406Povetacicept	Phase 1	Locked · track to reveal	Completed	136 est.	19 Aug 2025
A Phase 1 Dose Escalation Study of VX-973 in Healthy ParticipantsNCT06615570VX-973	Phase 1	Locked · track to reveal	Completed	30 est.	21 Jul 2025
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseaseNCT03745287CTX001	Phase 2	Locked · track to reveal	Completed	63 est.	7 Jul 2025
A Study Evaluating Efficacy and Safety of VX-993 for Acute Pain After a BunionectomyNCT06619847VX-993	Phase 2	Locked · track to reveal	Completed	367 est.	27 May 2025
A Study to Evaluate How the Body Processes 14C-VX-993 After a Single Oral DoseNCT0688626914C-VX-993	Phase 1	Locked · track to reveal	Completed	8 est.	17 May 2025

View all trials259

Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants

Phase 1RecruitingNCT07378865 ↗

Primary completion: Feb 2027; estimated
Enrollment: 12; estimated
Start: Feb 2026; Vertex Pharmaceuticals Incorporated

Conditions

Pain

Interventions

Suzetrigine

Brief summary

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. In addition, the purpose is also to evaluate the relative infant dose (RID), safety and tolerability of SUZ and its active metabolite in lactating female…

Full record on ClinicalTrials.gov ↗

Change timeline

1 recorded · latest 1 Mar 2026

Status change · 1

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Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Phase 3Active, not recruitingNCT05331183 ↗

Primary completion: Apr 2027; estimated
Enrollment: 297; estimated
Start: Nov 2022; Vertex Pharmaceuticals Incorporated

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVA
IVA

Brief summary

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 (CFTR) gene mutation.

Full record on ClinicalTrials.gov ↗

Change timeline

6 recorded · latest 1 Jul 2024

Status change · 3

Completion date move · 2

Enrollment change · 1

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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Phase 3Active, not recruitingNCT05329649 ↗

Primary completion: Jun 2027; estimated
Enrollment: 13; estimated
Start: May 2022; Vertex Pharmaceuticals Incorporated

Conditions

Sickle Cell Disease
Hydroxyurea Failure
Hydroxyurea Intolerance
Hemoglobinopathies
Hematological Diseases

Interventions

CTX001

Brief summary

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 hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Full record on ClinicalTrials.gov ↗

Change timeline

3 recorded · latest 1 Apr 2026

Enrollment change · 2

Status change · 1

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Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Phase 3RecruitingNCT05477563 ↗

Primary completion: Jun 2027; estimated
Enrollment: 26; estimated
Start: Aug 2022; Vertex Pharmaceuticals Incorporated

Conditions

Beta-Thalassemia
Thalassemia
Hematologic Diseases
Genetic Diseases, Inborn
Hemoglobinopathies
Sickle Cell Disease
Sickle Cell Anemia

Interventions

CTX001

Brief summary

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Full record on ClinicalTrials.gov ↗

Change timeline

3 recorded · latest 1 Jul 2025

Enrollment change · 2

Completion date move · 1

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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Phase 3Active, not recruitingNCT05356195 ↗

Primary completion: Nov 2027; estimated
Enrollment: 16; estimated
Start: May 2022; Vertex Pharmaceuticals Incorporated

Conditions

Beta-Thalassemia
Thalassemia
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

Interventions

CTX001

Brief summary

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).

Full record on ClinicalTrials.gov ↗

Change timeline

3 recorded · latest 1 Apr 2026

Enrollment change · 2

Status change · 1

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RUBY-3: An Open-label Study of Povetacicept in Autoantibody-Associated Glomerular Diseases

Phase 1Active, not recruitingNCT05732402 ↗

Primary completion: Mar 2028; estimated
Enrollment: 72; estimated
Start: Mar 2023; Alpine Immune Sciences, Inc.

Conditions

Lupus Nephritis
Immunoglobulin A Nephropathy
Membranous Nephropathy
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Interventions

Povetacicept

Brief summary

The goal of this clinical study is to evaluate multiple dose levels of povetacicept in adults with immunoglobulin A (IgA) nephropathy (IgAN), primary membranous nephropathy (pMN), lupus-related kidney disease (lupus nephritis - LN), or anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis (AAV) to…

Full record on ClinicalTrials.gov ↗

Change timeline

5 recorded · latest 1 Nov 2025

Enrollment change · 3

Completion date move · 1

Status change · 1

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Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Phase 3Active, not recruitingNCT05422222 ↗

Primary completion: Jun 2030; estimated
Enrollment: 210; estimated
Start: Jun 2022; Vertex Pharmaceuticals Incorporated

Conditions

Cystic Fibrosis

Interventions

VX-121/TEZ/D-IVA

Brief summary

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) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Full record on ClinicalTrials.gov ↗

Change timeline

3 recorded · latest 1 Oct 2024

Status change · 3

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A Long-term Follow-up Study in Participants Who Received CTX001

Phase 3Enrolling by invitationNCT04208529 ↗

Primary completion: Sept 2039; estimated
Enrollment: 160; estimated
Start: Jan 2021; Vertex Pharmaceuticals Incorporated

Conditions

Beta-Thalassemia
Thalassemia
Sickle Cell Disease
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Sickle Cell Anemia

Interventions

CTX001

Brief summary

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 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or…

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.

Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis

Phase 1Active, not recruitingNCT06154447 ↗

Primary completion: Jul 2026; estimated
Enrollment: 165; estimated
Start: Dec 2023; Vertex Pharmaceuticals Incorporated

Conditions

Cystic Fibrosis

Interventions

VX-828
Placebo
Itraconazole
Midazolam
Tezacaftor
VX-118
Placebo
Deutivacaftor

Brief summary

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 combination with D-IVA with or without TEZ in participants with cystic fibrosis (CF).

Full record on ClinicalTrials.gov ↗

Change timeline

6 recorded · latest 1 Jun 2026

Completion date move · 3

Enrollment change · 2

Status change · 1

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RUBY-4: An Open-label Study of Povetacicept in Participants With Autoimmune Cytopenias

Phase 1Active, not recruitingNCT05757570 ↗

Primary completion: Jul 2026; estimated
Enrollment: 30; estimated
Start: Jul 2023; Alpine Immune Sciences, Inc.

Conditions

Immune Thrombocytopenia
Idiopathic Thrombocytopenic Purpura
Warm Autoimmune Hemolytic Anemia
Cold Agglutinin Disease

Interventions

povetacicept

Brief summary

The goal of this clinical study is to evaluate povetacicept in adults with autoimmune cytopenias of immune thrombocytopenia, autoimmune hemolytic anemia, and cold agglutinin disease to determine if povetacicept is safe and potentially beneficial in treating these diseases. During the study treatment period…

Full record on ClinicalTrials.gov ↗

Change timeline

5 recorded · latest 1 Aug 2025

Enrollment change · 2

Status change · 2

Completion date move · 1

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A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Phase 1Active, not recruitingNCT05668741 ↗

Primary completion: Apr 2026; actual
Enrollment: 26; estimated
Start: Feb 2023; Vertex Pharmaceuticals Incorporated

Conditions

Cystic Fibrosis

Interventions

VX-522 mRNA therapy
IVA

Brief summary

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

Full record on ClinicalTrials.gov ↗

Change timeline

11 recorded · latest 1 Jun 2026

Status change · 4

Completion date move · 4

Enrollment change · 3

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Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants

Phase 1CompletedNCT07349394 ↗

Primary completion: Feb 2026; actual
Enrollment: 18; estimated
Start: Jan 2026; Vertex Pharmaceuticals Incorporated

Conditions

Cystic Fibrosis

Interventions

VNZ/TEZ/D-IVA
Rosuvastatin

Brief summary

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 rosuvastatin.

Full record on ClinicalTrials.gov ↗

Change timeline

5 recorded · latest 1 May 2026

Status change · 3

Completion date move · 1

Enrollment change · 1

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Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants

Phase 1CompletedNCT07074327 ↗

Primary completion: Feb 2026; actual
Enrollment: 74; estimated
Start: Jul 2025; Vertex Pharmaceuticals Incorporated

Conditions

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Interventions

VX-407
LNG/EE
NGM/EE
NET/EE
DRSP/EE

Brief summary

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. Also, to evaluate the safety and tolerability of co-administration of VX-407 with LNG/EE,…

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Apr 2026

Status change · 2

Enrollment change · 1

Completion date move · 1

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A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Phase 2CompletedNCT03655678 ↗

Primary completion: Nov 2025; actual
Enrollment: 59; estimated
Start: Sept 2018; Vertex Pharmaceuticals Incorporated

Conditions

Beta-Thalassemia
Thalassemia
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

Interventions

CTX001

Brief summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Jan 2026

Completion date move · 2

Status change · 1

Enrollment change · 1

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A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Phase 2CompletedNCT03745287 ↗

Primary completion: Jul 2025; actual
Enrollment: 63; estimated
Start: Nov 2018; Vertex Pharmaceuticals Incorporated

Conditions

Sickle Cell Disease
Hematological Diseases
Hemoglobinopathies

Interventions

CTX001

Brief summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Sept 2025

Completion date move · 2

Status change · 1

Enrollment change · 1

Track Vertex Pharmaceuticals drugs to see every change the night it lands.

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