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Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden

NCT ID: NCT03263091

Last Updated: 2024-08-01

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

184 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-29

Study Completion Date

2023-06-20

Brief Summary

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The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden.

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Detailed Description

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This study includes an Open-Label Lead in, a Double-Blind component, and an Open-Label High Erythropoietin component. There is a screening period of up to 42 days followed by a treatment period of 52 weeks and a 4-week end of treatment assessment.

Conditions

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Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Roxadustat

Open-label, lead-in: Participants will receive sequential escalating roxadustat doses (1.5 milligrams/kilograms \[mg/kg\], 2.0 mg/kg and 2.5 mg/kg), three times a week (TIW) based upon their actual weight at the randomization visit to identify the starting dose for double-blind period.

Double-blind: Participants will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks.

Open-label: Participants with high serum erythropoietin levels (\>400 milli-international units \[mIU\]/milliliter \[mL\] mIU/mL) will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks.

Group Type EXPERIMENTAL

Roxadustat

Intervention Type DRUG

Oral tablets

Placebo

Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Oral tablets

Interventions

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Roxadustat

Oral tablets

Intervention Type DRUG

Placebo

Oral tablets

Intervention Type DRUG

Other Intervention Names

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FG-4592 ASP1517 AZD9941

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of primary MDS classified by the International Prognostic Scoring System - Revised (IPSS-R) as very low, low or intermediate risk with \<5% bone marrow blasts. There is no minimum time from diagnosis to registration/randomization except to allow for proper IPSS-R classification to be made (within 16 weeks prior to randomization), and to show transfusion dependence for participants in both portions of the study.
* RBC transfusion of either 2-4 pRBC units during the 8 weeks prior to registration/randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to registration/randomization. Open-Label Lead-in participants only, the requirement to demonstrate transfusion dependence can also be met by a Principal Investigator starting this particular participant on pRBC transfusion during the screening period.
* No restriction on prior use of recombinant erythropoietins or analogues (erythropoiesis-stimulating agents \[ESAs\]), except no ESA use within 8 weeks prior to Day 1 registration/randomization.
* Hemoglobin (Hb) ≤10.0 grams/deciliter (g/dL) during screening
* Eastern Cooperative Oncology Group (ECOG) of 0-2 at screening

Exclusion Criteria

* Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation therapy (\>25% of bone marrow reserve), and or/other significant chemical or radiation exposure
* Significant myelofibrosis (\>2+ fibrosis)
* MDS associated with 5q(del) cytogenetic abnormality
* Screen serum erythropoietin level \> 400 milli-international units (mIU)/milliliter (mL) • Clinically significant anemia, as determined by the investigator, due to non-MDS etiologies such as iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis or anemia or hemorrhage or hereditary anemia such as sickle cell anemia or thalassemia.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AstraZeneca

INDUSTRY

Sponsor Role collaborator

Astellas Pharma Inc

INDUSTRY

Sponsor Role collaborator

FibroGen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Investigational Site

Philadelphia, Pennsylvania, United States

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Charleston, South Carolina, United States

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Houston, Texas, United States

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Charlottesville, Virginia, United States

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Darlinghurst, New South Wales, Australia

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Bakersfield, California, United States

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Beverly Hills, California, United States

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Burbank, California, United States

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Encino, California, United States

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Laguna Hills, 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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Oceanside, California, United States

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Pasadena, California, United States

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Santa Maria, California, United States

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Torrance, California, United States

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Valencia, California, United States

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Ventura, California, United States

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Westlake Village, California, United States

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Whittier, California, United States

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Grand Junction, Colorado, United States

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Pembroke Pines, Florida, United States

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Weston, Florida, United States

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Atlanta, Georgia, United States

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Augusta, Georgia, United States

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St Louis, Missouri, United States

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Charlotte, North Carolina, United States

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Canton, Ohio, United States

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Cleveland, Ohio, United States

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Cleveland, Ohio, United States

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Mayfield Heights, Ohio, United States

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Liverpool, New South Wales, Australia

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South Brisbane, Queensland, Australia

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Victoria Park, Saint Albans, Australia

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Hobart, Tasmania, Australia

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Wilrijk, Antwerpen, Belgium

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Brussels, Brussels Capital, Belgium

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Hasselt, Limburg, Belgium

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Bruges, West-Vlaanderen, Belgium

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Brussels, , Belgium

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Yvoir, , Belgium

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Vancouver, British Columbia, Canada

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London, Ontario, Canada

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Odense, , Denmark

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Nice, Alpes-Maritimes, France

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Grenoble, Isère, France

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Paris, , France

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Tours, , France

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Freiburg im Breisgau, Baden-Wurttemberg, Germany

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München, Bavaria, Germany

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Münster, North Rhine-Westphalia, Germany

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Dresden, Saxony, Germany

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Leipzig, Saxony, Germany

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Düsseldorf, , Germany

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Düsseldorf, , Germany

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Vellore, Tamil Nadu, India

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Kolkata, West Bengal, India

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Kfar Saba, Central District, Israel

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Ẕerifin, Central District, Israel

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Nahariya, Northern District, Israel

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Haifa, , Israel

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Ramat Gan, , Israel

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Tel Aviv, , Israel

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Tel Litwinsky, , Israel

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Monza, Lombardy, Italy

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Meldola, Ravenna, Italy

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Alessandria, , Italy

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Bologna, , Italy

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Florence, , Italy

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Genova, , Italy

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Lecce, , Italy

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Milan, , Italy

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Reggio Calabria, , Italy

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Rimini, , Italy

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Roma, , Italy

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Terni, , Italy

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Torino, , Italy

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Varese, , Italy

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Bialystok, , Poland

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Bydgoszcz, , Poland

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Krakow, , Poland

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Piła, , Poland

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Skorzewo, , Poland

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Słupsk, , Poland

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Warsaw, , Poland

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Kaluga, Russian Federation, Russia

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Moscow, Russian Federation, Russia

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Moscow, Russian Federation, Russia

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Moscow, Russian Federation, Russia

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Omsk, Russian Federation, Russia

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Saint Petersburg, Russian Federation, Russia

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Saint Petersburg, Russian Federation, Russia

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Saint Petersburg, Russian Federation, Russia

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Saint Petersburg, , Russia

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Incheon, Incheon Gwang'yeogsi, South Korea

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Incheon, Incheon Gwangyeogsi, South Korea

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Hwasun, Jeonranamdo, South Korea

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Seoul, Seoul Teugbyeolsi, South Korea

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Seoul, , South Korea

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Sabadell, Barcelona, Spain

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Barcelona, Catalonia, Spain

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Pamplona, Navarre, Spain

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Barcelona, , Spain

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Barcelona, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Salamanca, , Spain

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Seville, , Spain

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Seville, , Spain

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Valencia, , Spain

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Ankara, , Turkey (Türkiye)

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Dikimevi, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Kayseri, , Turkey (Türkiye)

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Tekirdağ, , Turkey (Türkiye)

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Yenişehir, , Turkey (Türkiye)

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Boston, Lincolnshire, United Kingdom

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Headington, Oxford, United Kingdom

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Harrow, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Countries

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United States Australia Belgium Canada Denmark France Germany India Israel Italy Poland Russia South Korea Spain Turkey (Türkiye) United Kingdom

References

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Henry DH, Glaspy J, Harrup R, Mittelman M, Zhou A, Carraway HE, Bradley C, Saha G, Modelska K, Bartels P, Leong R, Yu KP. Roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndrome: Open-label, dose-selection, lead-in stage of a phase 3 study. Am J Hematol. 2022 Feb 1;97(2):174-184. doi: 10.1002/ajh.26397. Epub 2021 Nov 9.

Reference Type DERIVED
PMID: 34724251 (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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FGCL-4592-082

Identifier Type: -

Identifier Source: org_study_id

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