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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-000185-42 | EudraCT Number |
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This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-147 | Experimental | All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-147 | Drug | Tablets for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in UPCR | From Baseline up to Week 13 |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 17 | |
| Maximum Observed Concentration (Cmax) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham - The Kirklin Clinic | Birmingham | Alabama | 35294 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36920755 | Derived | Egbuna O, Zimmerman B, Manos G, Fortier A, Chirieac MC, Dakin LA, Friedman DJ, Bramham K, Campbell K, Knebelmann B, Barisoni L, Falk RJ, Gipson DS, Lipkowitz MS, Ojo A, Bunnage ME, Pollak MR, Altshuler D, Chertow GM; VX19-147-101 Study Group. Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants. N Engl J Med. 2023 Mar 16;388(11):969-979. doi: 10.1056/NEJMoa2202396. | |
| 33767059 |
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Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.
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This study was conducted on adult participants who had APOL1-mediated focal segmental glomerulosclerosis (FSGS).
This study was planned in 2 parts: Part A (Treatment Period), which consisted of 2 cohorts (i.e., cohort 1 and 2) and Part B (Optional Exploratory Off-treatment Follow-up Period). The primary and secondary efficacy analyses and safety analyses were planned for only Part A.
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| ID | Title | Description |
|---|---|---|
| FG000 | VX-147 | All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 26, 2021 | Apr 28, 2023 |
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| Observed Pre-dose Concentration (Ctrough) of VX-147 | Pre-dose on Day 8, 15, Week 3, 5, 9 and 13 |
| Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5 |
| California Institute of Renal Research |
| San Diego |
| California |
| 92123 |
| United States |
| The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension | Washington D.C. | District of Columbia | 20037 | United States |
| Howard University Hospital | Washington D.C. | District of Columbia | 20060 | United States |
| Kidney and Hypertension Specialists of Miami | Miami | Florida | 33150 | United States |
| Florida Premier Research Institute | Winter Park | Florida | 32789 | United States |
| Morehouse School of Medicine, Grady Memorial Hospital | Atlanta | Georgia | 30310 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | United States |
| Georgia Nehphrology | Lawrenceville | Georgia | 30046 | United States |
| Central Georgia Kidney Specialists PC | Macon | Georgia | 31201 | United States |
| Renal Associates of Baton Rouge | Baton Rouge | Louisiana | 70808 | United States |
| Ochsner Medical Center - New Orleans | New Orleans | Louisiana | 70121 | United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | United States |
| Renal and Transplant Associates of New England, PC | Springfield | Massachusetts | 01107 | United States |
| Paragon Health, PC d/b/a Nephrology Center, PC | Kalamazoo | Michigan | 49007 | United States |
| Nephrology and Hypertension Associates, LTD | Tupelo | Mississippi | 38801 | United States |
| St Louis Kidney Care | St Louis | Missouri | 63136 | United States |
| Nevada Kidney Disease and Hypertension Centers | Las Vegas | Nevada | 89106 | United States |
| SUNY Downstate | Brooklyn | New York | 11203 | United States |
| Urban Family Practice | Buffalo | New York | 14201 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| UNC Kidney Center Division of Nephrology & Hypertension | Chapel Hill | North Carolina | 27599 | United States |
| Tryon Medical Partners | Charlotte | North Carolina | 28210 | United States |
| Durham Nephrology Associates, PA | Durham | North Carolina | 27704 | United States |
| Duke University School of Medicine - Duke Molecular Physiology Institute | Durham | North Carolina | 27710 | United States |
| Temple University Hospital | Philadelphia | Pennsylvania | 19140 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| South Carolina Nephrology and Hypertension Center, Inc. | Orangeburg | South Carolina | 29118 | United States |
| Vanderbilt University VU | Nashville | Tennessee | 37232 | United States |
| Prolato Clinical Research Center | Houston | Texas | 77054 | United States |
| Privia Medical Group | Houston | Texas | 77095 | United States |
| Hopital Henri Mondor | Créteil | France |
| Hôpital Bicêtre AP-HP | Le Kremlin-Bicêtre | France |
| Bichat Hospital | Paris | France |
| Hopitaux Universitaires Est Parisien - Hopital Tenon | Paris | France |
| Service de Nephrologie - Hopital Universitaire Necker | Paris | France |
| Université Paris-Descartes / Hôpital Européen Georges Pompidou | Paris | France |
| GCM Medical Group, PSC | San Juan | 00917 | Puerto Rico |
| University Hospitals of Leicester NHS Trust - Leicester General Hospital | Leicester | United Kingdom |
| Barts Health NHS Trust | London | United Kingdom |
| The Medicines Evaluation Unit | Manchester | United Kingdom |
| King's College Hospital NHS Foundation Trust - Guthrie Clinic | Southwark | United Kingdom |
| Derived |
| Bruggeman LA, Sedor JR, O'Toole JF. Apolipoprotein L1 and mechanisms of kidney disease susceptibility. Curr Opin Nephrol Hypertens. 2021 May 1;30(3):317-323. doi: 10.1097/MNH.0000000000000704. |
| Cohort 1 | The milestone represents the number of participants in Cohort 1. |
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| Cohort 2 | The milestone represents the number of participants in Cohort 2. |
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| COMPLETED |
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| NOT COMPLETED |
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All participants who received at least one dose of the study drug during the treatment period were included in the baseline analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | VX-147 | All participants received VX-147 at a dosage of 15 mg qd for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately ≥3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | The Baseline data were planned to be presented separately for Cohort 1 and Cohort 2. Here, "Number Analyzed" signifies participants who were evaluable for specified Cohort of the study. | Mean | Standard Deviation | years |
| ||||||||||||||||
| Sex: Female, Male | The Baseline data were planned to be presented separately for Cohort 1 and Cohort 2. Here, "Number Analyzed" signifies participants who were evaluable for specified Cohort of the study. | Count of Participants | Participants |
| |||||||||||||||||
| Ethnicity (NIH/OMB) | The Baseline data were planned to be presented separately for Cohort 1 and Cohort 2. Here, "Number Analyzed" signifies participants who were evaluable for specified Cohort of the study. | Count of Participants | Participants |
| |||||||||||||||||
| Race (NIH/OMB) | The Baseline data were planned to be presented separately for Cohort 1 and Cohort 2. Here, "Number Analyzed" signifies participants who were evaluable for specified Cohort of the study. | Count of Participants | Participants |
| |||||||||||||||||
| Urine Protein to Creatinine Ratio (UPCR) | The baseline data were planned to be presented for the overall treatment arm (i.e. Cohort 1 and Cohort 2). Here, "Number Analyzed" signifies participants who were evaluable for specified Cohort of the study. | Mean | Standard Deviation | g/g |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in UPCR | The Full Analysis Set (FAS) included all participants who received at least 1 dose of study drug in the treatment period. Here "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome and the "Number Analyzed" signifies participants who were evaluable for the specified Cohorts. | Posted | Geometric Mean | 95% Confidence Interval | percent change | From Baseline up to Week 13 |
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| ||||||||||||||||||||||||||||
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety set included all participants who received at least 1 dose of study drug in the treatment period. Here, the "Number Analyzed" signifies participants who were evaluable for the specified cohorts. | Posted | Count of Participants | Participants | From Baseline up to Week 17 |
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| ||||||||||||||||||||||||||||||
| Secondary | Maximum Observed Concentration (Cmax) of VX-147 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here "Number Analyzed" signifies participants who were evaluable at specified timepoint. | Posted | Mean | Standard Deviation | micrograms per milliliter (mcg/ml) | Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5 |
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| Secondary | Observed Pre-dose Concentration (Ctrough) of VX-147 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here "Number Analyzed" signifies participants who were evaluable at specified timepoint. | Posted | Mean | Standard Deviation | mcg/ml | Pre-dose on Day 8, 15, Week 3, 5, 9 and 13 |
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| Secondary | Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here "Number Analyzed" signifies participants who were evaluable at specified timepoint. | Posted | Mean | Standard Deviation | hour*mcg/mL (h*mcg/mL) | Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5 |
|
|
Day 1 up to Week 17
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | VX-147 : Cohort 1 | Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately ≥3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). | 0 | 3 | 0 | 3 | 3 | 3 |
| EG001 | VX-147: Cohort 2 | Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2 | 0 | 13 | 1 | 13 | 12 | 13 |
| EG002 | VX-147 Total | All participants received VX-147 at a dosage of 15 mg qd for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately ≥3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2. | 0 | 16 | 1 | 16 | 15 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 24.1 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | Meddra 24.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain lower | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Peripheral swelling | General disorders | MedDRA 24.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Tinea pedis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Tooth abscess | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Vaccination complication | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Blood bicarbonate decreased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Electrocardiogram ST segment depression | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Gamma-glutamyltransferase increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Transaminases increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Depressed mood | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Thinking abnormal | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Pollakiuria | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 20, 2021 | Apr 28, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D005923 | Glomerulosclerosis, Focal Segmental |
| ID | Term |
|---|---|
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
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|
| Male |
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| Cohort 2 |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Cohort 2 |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Cohort 2 |
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| Cohort 2 |
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| Total |
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