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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
Trial ID: NCT03263091
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.
A Phase 3 Randomized Double-Blind Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Patients With Lower Risk Myelodysplastic Syndrome (MDS) With Low Red Blood Cell (RBC) Transfusion Burden (LTB)
Key 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
- 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 Criteria:
- Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation
therapy (>25% of bone marrow reserve), and or/other significant chemical or radiation
- 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.
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