Status:

TERMINATED

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

Lead Sponsor:

FibroGen

Collaborating Sponsors:

AstraZeneca

Astellas Pharma Inc

Conditions:

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

Anemia

Eligibility:

All Genders

18+ years

Phase:

PHASE3

Brief Summary

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.

Detailed Description

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 ...

Eligibility Criteria

Inclusion

  • Key
  • 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
  • Key

Exclusion

  • 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.

Key Trial Info

Start Date :

January 29 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 20 2023

Estimated Enrollment :

184 Patients enrolled

Trial Details

Trial ID

NCT03263091

Start Date

January 29 2018

End Date

June 20 2023

Last Update

August 1 2024

Active Locations (124)

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Page 1 of 31 (124 locations)

1

Investigational Site

Bakersfield, California, United States, 93309

2

Investigational Site

Beverly Hills, California, United States, 90212

3

Investigational Site

Burbank, California, United States, 90212

4

Investigational Site

Encino, California, United States, 91436