Safety and Efficacy Study of Roxadustat to Treat Anemia in Patients With Chronic Kidney Disease, on Dialysis.

NCT ID: NCT02174731

Last Updated: 2019-12-16

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

2133 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-01

Study Completion Date

2018-09-26

Brief Summary

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The purpose of this study is to evaluate the efficacy and safety of roxadustat compared to epoetin alfa for the treatment of anemia in chronic kidney disease patients on dialysis.

Detailed Description

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This is a Phase 3, multicenter, randomized, open-label, active-controlled study to evaluate the efficacy and safety of roxadustat compared to epoetin alfa for the treatment of anemia in dialysis patients. Patients on hemodialysis (HD) or peritoneal dialysis (PD) who have been treated with an erythropoietin analogue or have an indication for treatment with an erythropoietin analogue will be evaluated for eligibility and randomized at a 1:1 ratio to treatment with roxadustat (with discontinuation of prior erythropoietin analogue therapy) or to an active-control group treated with epoetin alfa

Conditions

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Anemia

Keywords

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Renal, CKD, Epoetin alfa, Roxadustat, dialysis, anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Roxadustat

Group Type EXPERIMENTAL

Roxadustat

Intervention Type DRUG

Roxadustat will be administered orally three times a week (TIW) to achieve an Hb level of 11 g/dL and maintain a Hb level of 11±1 g/dL.

Epoetin alfa

Group Type ACTIVE_COMPARATOR

Epoetin alfa

Intervention Type DRUG

Epoetin alfa will be administered TIW consistent with approved prescribing information for epoetin alfa to achieve an Hb level of 11 g/dL and maintain a Hb level of 11±1 g/dL.

Interventions

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Roxadustat

Roxadustat will be administered orally three times a week (TIW) to achieve an Hb level of 11 g/dL and maintain a Hb level of 11±1 g/dL.

Intervention Type DRUG

Epoetin alfa

Epoetin alfa will be administered TIW consistent with approved prescribing information for epoetin alfa to achieve an Hb level of 11 g/dL and maintain a Hb level of 11±1 g/dL.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Provision of Informed Consent prior to any study specific procedures
2. Age ≥18 years at screening visit 1
3. Previous versions of the protocol prior to US amendment ver 6.0 and outside of US amendment ver 7.0:

Receiving or initiating hemodialysis or peritoneal dialysis for treatment of native kidney end-stage renal disease (ESRD) at least 30 days prior to visit 1. Patients treated with hemodialysis must have access consisting of an arteriovenous fistula, AV graft, or tunneled (permanent) catheter. Patients on peritoneal dialysis must have a functioning peritoneal dialysis catheter in place.

Starting with US amendment ver. 6.0 and outside of US amendment ver 7.0 (changed to recruit incident dialysis patients only):

Receiving or initiating hemodialysis or peritoneal dialysis for treatment of native kidney end-stage renal disease (ESRD) for a minimum of 2 weeks and a maximum of 4 months prior to randomization. Patients treated with hemodialysis must have access consisting of an arteriovenous fistula, AV graft, or tunneled (permanent) catheter. Patients on peritoneal dialysis must have a functioning peritoneal dialysis catheter in place.
4. Two central laboratory Hb values during the screening period, obtained at least 7 days apart, must be \<12 g/dL in patients currently treated with an erythropoietin analogue or \<10 g/dL in patients not currently treated with an erythropoietin analogue. Patients are considered not currently treated if they have not received either Mircera® for at least 8 weeks or any other erythropoietin analogue for at least 4 weeks prior to visit 1.
5. Ferritin ≥100 ng/mL at randomization (obtained from screening visit)
6. TSAT ≥20% at randomization (obtained from screening visit)
7. Serum folate level ≥ lower limit of normal (LLN) at randomization (obtained from screening visit)
8. Serum vitamin B12 level ≥ LLN at randomization (obtained from screening visit)
9. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3x upper limit of normal (ULN), and total bilirubin (Tbili) ≤1.5 x ULN at randomization (obtained from screening visit)
10. Body weight 45 to 160 kg (prescribed dry weight)

Exclusion Criteria

1. Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site)
2. Previous randomisation in the present study
3. New York Heart Association Class III or IV congestive heart failure at enrolment
4. Myocardial infarction, acute coronary syndrome, stroke, seizure or a thrombotic/thromboembolic event (e.g., deep vein thrombosis or pulmonary embolism) within 12 weeks prior to randomization
5. History of chronic liver disease (e.g., chronic infectious hepatitis, chronic auto-immune liver disease, cirrhosis or fibrosis of the liver)
6. Known hereditary hematologic disease such as thalassemia, sickle cell anemia, a history of pure red cell aplasia or other known causes for anemia other than CKD
7. Known and untreated retinal vein occlusion or known and untreated proliferative diabetic retinopathy (risk for retinal vein thrombosis)
8. Diagnosis or suspicion (e.g. complex kidney cyst of Bosniak Category IIF, III or IV) of renal cell carcinoma on renal ultrasound (or other imaging procedure e.g. CT scan or MRI) conducted at screening or within 12 weeks prior to randomization.
9. Uncontrolled hypertension at the time of randomization (defined as systolic BP ≥180 mmHg or diastolic BP ≥100 mmHg on repeated measurement post-dialysis in hemodialysis patients or at any time in peritoneal dialysis patients), contraindication to epoetin alfa treatment (e.g., pure red cell aplasia, hypersensitivity or know inability to tolerate epoetin alfa)
10. History of prostate cancer, breast cancer or any other malignancy, except the following: cancers determined to be cured or in remission for ≥5 years, curatively resected basal cell or squamous cell skin cancers, cervical cancer in situ or resected colonic polyps.
11. Positive for any of the following: human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) or anti-hepatitis C virus antibody (anti-HCV Ab)
12. Chronic inflammatory diseases such as rheumatoid arthritis, SLE, ankylosing spondylitis, psoriatic arthritis or inflammatory bowel disease that is determined to be the principal cause of anemia
13. Known hemosiderosis, hemochromatosis or hypercoagulable condition
14. Any prior organ transplant with the exception of an autologous renal transplant or a renal transplant that was subsequently removed ("explanted") or scheduled organ transplantation date
15. Any red blood cell (RBC) transfusion during the screening period
16. Any current condition leading to active significant blood loss
17. Any prior treatment with roxadustat or a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI)
18. Has received another new chemical entity (defined as a compound which has not been approved for marketing) or has participated in any other clinical study that included drug treatment within the month preceding the first administration of IP in this study. (Note: patients consented and screened, but not randomized in this study or a previous study are not excluded)
19. History of alcohol or drug abuse within 2 years prior to randomization
20. Females of childbearing potential, unless using contraception as detailed in the protocol or sexual abstinence (see Section 3.8)
21. Pregnant or breastfeeding females
22. Known allergy to the investigational product or any of its ingredients
23. Any medical condition, including active, clinically significant infection, that in the opinion of the investigator or Sponsor may pose a safety risk to a patient in this study, which may confound efficacy or safety assessment, or may interfere with study participation
Minimum Eligible Age

18 Years

Maximum Eligible Age

130 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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FibroGen

INDUSTRY

Sponsor Role collaborator

AstraZeneca

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Steven Fishbane, MD

Role: PRINCIPAL_INVESTIGATOR

Chief Division of Kidney Diseases and Hypertension, North Shore University Hospital, Great Neck, NY, USA

Mark Houser, MD

Role: STUDY_DIRECTOR

AZ R&D, Gaithersburg, USA

Locations

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Huntsville, Alabama, United States

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El Centro, California, United States

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Fairfield, California, United States

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Glendale, California, United States

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Long Beach, California, United States

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Long Beach, California, United States

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Los Angeles, California, United States

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Los Angeles, California, United States

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Lynwood, California, United States

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Northridge, California, United States

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Ontario, California, United States

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Orange, California, United States

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Riverside, California, United States

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Sacramento, California, United States

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Sun Valley, California, United States

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Whittier, California, United States

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Whittier, California, United States

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Denver, Colorado, United States

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New Haven, Connecticut, United States

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Miami Gardens, Florida, United States

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River Forest, Illinois, United States

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Wichita, Kansas, United States

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Baton Rouge, Louisiana, United States

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Lanham, Maryland, United States

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Plymouth, Massachusetts, United States

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Roseville, Michigan, United States

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Gulfport, Mississippi, United States

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Kansas City, Missouri, United States

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Reno, Nevada, United States

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Paterson, New Jersey, United States

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Great Neck, New York, United States

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Orchard Park, New York, United States

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The Bronx, New York, United States

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Asheville, North Carolina, United States

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Charlotte, North Carolina, United States

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Wilmington, North Carolina, United States

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Cincinnati, Ohio, United States

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Shawnee, Oklahoma, United States

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Bethlehem, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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East Providence, Rhode Island, United States

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Austin, Texas, United States

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Austin, Texas, United States

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Houston, Texas, United States

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Houston, Texas, United States

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San Antonio, Texas, United States

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San Antonio, Texas, United States

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San Antonio, Texas, United States

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Newport News, Virginia, United States

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Sterling, Virginia, United States

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Bluefield, West Virginia, United States

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Camperdown, , Australia

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Clayton, , Australia

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Concord, , Australia

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Launceston, , Australia

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Prahan, , Australia

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St Leonards, , Australia

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Wahroonga, , Australia

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Botevgrad, , Bulgaria

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Dupnitsa, , Bulgaria

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Gotse Delchev, , Bulgaria

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Haskovo, , Bulgaria

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Razlog, , Bulgaria

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Samokov, , Bulgaria

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Sandanski, , Bulgaria

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Shumen, , Bulgaria

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Silistra, , Bulgaria

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Sliven, , Bulgaria

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Smolyan, , Bulgaria

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Yambol, , Bulgaria

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Edmonton, Alberta, Canada

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Kamloops, British Columbia, Canada

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Saint John, New Brunswick, Canada

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Sydney, Nova Scotia, Canada

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Brampton, Ontario, Canada

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Oshawa, Ontario, Canada

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Scarborough Village, Ontario, Canada

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Toronto, Ontario, Canada

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Greenfield Park, Quebec, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Québec, Quebec, Canada

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Brno, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Rychnov nad Kněžnou, , Czechia

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Slaný, , Czechia

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Tábor, , Czechia

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Ústí nad Labem, , Czechia

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Ajka, , Hungary

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Budapest, , Hungary

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Budapest, , Hungary

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Budapest, , Hungary

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Debrecen, , Hungary

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Miskolc, , Hungary

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Nagykanizsa, , Hungary

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Székesfehérvár, , Hungary

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Tatabánya, , Hungary

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Chennai, , India

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Ghaziabad NCR, , India

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Hyderabad, , India

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Kolkata, , India

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Lucknow, , India

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Maharashtra, , India

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Mumbai, , India

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Mysore, , India

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Nadiād, , India

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New Delhi, , India

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New Delhi, , India

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Pune, , India

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Vellore, , India

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Vijayawada, , India

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Aguascalientes, , Mexico

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Guadalajara, , Mexico

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México, , Mexico

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Veracruz, , Mexico

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Lima, , Peru

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Lima, , Peru

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Lima, , Peru

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Cebu City, , Philippines

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Iloilo City, , Philippines

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Ciechanów, , Poland

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Gmina Końskie, , Poland

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Katowice, , Poland

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Lodz, , Poland

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Lodz, , Poland

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Pszczyna, , Poland

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Suwałki, , Poland

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Tarnów, , Poland

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Warsaw, , Poland

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Arkhangelsk, , Russia

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Barnaul, , Russia

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Irkutsk, , Russia

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Izhevsk, , Russia

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Kemerovo, , Russia

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Moscow, , Russia

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Moscow, , Russia

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Nizhny Novgorod, , Russia

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Penza, , Russia

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Perm, , Russia

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Pyatigorsk, , Russia

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Ryazan, , Russia

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Saint Petersburg, , Russia

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Ufa, , Russia

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Banská Bystrica, , Slovakia

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Banská Bystrica, , Slovakia

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Bratislava, , Slovakia

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Bratislava, , Slovakia

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Košice, , Slovakia

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Kráľovský Chlmec, , Slovakia

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Nové Zámky, , Slovakia

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Zvolen, , Slovakia

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Almería, , Spain

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Barcelona, , Spain

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Granollers (Barcelona), , Spain

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Málaga, , Spain

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Seville, , Spain

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Seville, , Spain

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Tarragona, , Spain

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Valencia, , Spain

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Karlstad, , Sweden

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Stockholm, , Sweden

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Uppsala, , Sweden

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Västerås, , Sweden

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Bangkok, , Thailand

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Bangkok, , Thailand

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Chernivtsi, , Ukraine

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Dnipro, , Ukraine

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Ivano-Frankivsk, , Ukraine

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Ivano-Frankivsk, , Ukraine

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Kyiv, , Ukraine

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Kyiv, , Ukraine

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Kyiv, , Ukraine

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Kyiv, , Ukraine

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Odesa, , Ukraine

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Odesa, , Ukraine

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Poltava, , Ukraine

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Ternopil, , Ukraine

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Uzhhorod, , Ukraine

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Vinnytsia, , Ukraine

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Zaporizhzhya, , Ukraine

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Zaporizhzhya, , Ukraine

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Zaporizhzhya, , Ukraine

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Can Tho, , Vietnam

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Hanoi, , Vietnam

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Hochiminh, , Vietnam

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Huế, , Vietnam

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Countries

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Argentina Brazil Romania United States Australia Bulgaria Canada Czechia Hungary India Mexico Peru Philippines Poland Russia Slovakia Spain Sweden Thailand Ukraine Vietnam

References

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Hamano T, Yamaguchi Y, Goto K, Martin S, Jiletcovici A, Dellanna F, Akizawa T, Barratt J. Risk Factors for Thromboembolic Events in Patients With Dialysis-Dependent CKD: Pooled Analysis of Four Global Roxadustat Phase 3 Trials. Adv Ther. 2024 Apr;41(4):1553-1575. doi: 10.1007/s12325-023-02728-2. Epub 2024 Feb 16.

Reference Type DERIVED
PMID: 38363466 (View on PubMed)

Barratt J, Dellanna F, Portoles J, Choukroun G, De Nicola L, Young J, Dimkovic N, Reusch M. Safety of Roxadustat Versus Erythropoiesis-Stimulating Agents in Patients with Anemia of Non-dialysis-Dependent or Incident-to-Dialysis Chronic Kidney Disease: Pooled Analysis of Four Phase 3 Studies. Adv Ther. 2023 Apr;40(4):1546-1559. doi: 10.1007/s12325-023-02433-0. Epub 2023 Feb 7.

Reference Type DERIVED
PMID: 36749544 (View on PubMed)

Natale P, Palmer SC, Jaure A, Hodson EM, Ruospo M, Cooper TE, Hahn D, Saglimbene VM, Craig JC, Strippoli GF. Hypoxia-inducible factor stabilisers for the anaemia of chronic kidney disease. Cochrane Database Syst Rev. 2022 Aug 25;8(8):CD013751. doi: 10.1002/14651858.CD013751.pub2.

Reference Type DERIVED
PMID: 36005278 (View on PubMed)

Fishbane S, Pollock CA, El-Shahawy M, Escudero ET, Rastogi A, Van BP, Frison L, Houser M, Pola M, Little DJ, Guzman N, Pergola PE. Roxadustat Versus Epoetin Alfa for Treating Anemia in Patients with Chronic Kidney Disease on Dialysis: Results from the Randomized Phase 3 ROCKIES Study. J Am Soc Nephrol. 2022 Apr;33(4):850-866. doi: 10.1681/ASN.2020111638.

Reference Type DERIVED
PMID: 35361724 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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D5740C00002

Identifier Type: -

Identifier Source: org_study_id