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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-517393-13-00 | Other Identifier | EU CTIS |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-407 | Experimental | Participants will receive VX-407 for up to 52 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-407 | Drug | Tablets for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| htTKV on MRI Over Time | Baseline up to End of Study (Week 52) |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 54 | |
| Maximum Observed Plasma Concentration (Cmax) of VX-407 | From Day 1 up to Week 52 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria will apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Contact | 617-341-6777 | medicalinfo@vrtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Alabama Kidney Research | Recruiting | Alabaster | Alabama | 35007 | United States | |
| UAB Nephrology Research at CH19 |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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| Area Under the Concentration Versus Time Curve (AUC) of VX-407 | From Day 1 up to Week 52 |
| Recruiting |
| Birmingham |
| Alabama |
| 35294 |
| United States |
| Kaiser Permanente Los Angeles Medical Center | Recruiting | Los Angeles | California | 90027 | United States |
| UCSF Clinical Research Center at Parnassus | Recruiting | San Francisco | California | 94143 | United States |
| Nephrology & Hypertension Associates, PC | Recruiting | Middlebury | Connecticut | 06762 | United States |
| Yale University - Yale Nephrology Clinical Research Clinic | Recruiting | New Haven | Connecticut | 06510 | United States |
| Mayo Clinic - Jacksonville | Recruiting | Jacksonville | Florida | 32224 | United States |
| Emory School of Medicine - Renal Division | Recruiting | Atlanta | Georgia | 30322 | United States |
| Renal Associates | Recruiting | Columbus | Georgia | 31904 | United States |
| UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology | Recruiting | Chicago | Illinois | 60637 | United States |
| University of Iowa Health Care Medical Center- Nephrology | Recruiting | Iowa City | Iowa | 52242 | United States |
| Clinical & Translational Science Unit (CTSU) - Nephrology | Recruiting | Kansas City | Kansas | 66160 | United States |
| University of Maryland Medical Center - General Clinical Research Center | Recruiting | Baltimore | Maryland | 21201 | United States |
| Tufts Medical Center | Recruiting | Boston | Massachusetts | 02111 | United States |
| Beth Israel Deaconess Medical Center - Nephrology | Recruiting | Boston | Massachusetts | 02215 | United States |
| University of Minnesota -Clinics and Surgery Center (CSC) - Endocrine Clinic | Recruiting | Minneapolis | Minnesota | 55455 | United States |
| Mayo Clinic - Rochester | Recruiting | Rochester | Minnesota | 55905 | United States |
| DaVita Kidney Care - Las Vegas | Recruiting | Las Vegas | Nevada | 89107 | United States |
| The Rogosin Kidney Center | Recruiting | New York | New York | 10021 | United States |
| General Nephrology Clinic - New York City | Recruiting | New York | New York | 10032 | United States |
| Cleveland Clinic - Main Campus | Recruiting | Cleveland | Ohio | 44195 | United States |
| Geisinger Clinic | Recruiting | Danville | Pennsylvania | 17822 | United States |
| Perelman Center for Advanced Medicine - Nephrology | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Nephrology Clinical Trials Center | Recruiting | Nashville | Tennessee | 37232 | United States |
| UVM Medical Center - 1 South Prospect Street | Recruiting | Burlington | Vermont | 05401 | United States |
| Swedish Center for Comprehensive Care | Recruiting | Seattle | Washington | 98104 | United States |
| Universite Catholique de Louvain - Nephrology | Recruiting | Brussels | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology | Recruiting | Leuven | Belgium |
| McGill University Health Centre | Recruiting | Montreal | Canada |
| Centre Hospitalier Regional Universitaire (CHRU) Brest | Recruiting | Brest | France |
| Hopital Necker Enfants Malades - Neurology | Recruiting | Paris | France |
| Hopital Bretonneau | Recruiting | Tours | France |
| Charité - Klinik für Nephrologie und Intensivmedizin der | Recruiting | Berlin | Germany |
| University Hospital Cologne (Koeln) - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin | Recruiting | Cologne | Germany |
| UMCG - Nephrology | Recruiting | Groningen | Netherlands |
| Fundacio Puigvert | Recruiting | Barcelona | Spain |
| Hospital Fundacion Jimenez Diaz | Recruiting | Madrid | Spain |
| Queen Elizabeth Hospital Birmingham | Recruiting | Birmingham | United Kingdom |
| King's College Hospital - Pulmonology | Recruiting | London | United Kingdom |
| Royal Free Hospital - Nephrology | Recruiting | London | United Kingdom |
| St George's Hospital London - Nephrology | Recruiting | London | United Kingdom |
| Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital - Nephrology | Recruiting | Newcastle upon Tyne | United Kingdom |
| Northern General Hospital | Recruiting | Sheffield | United Kingdom |
| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
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