Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)
About this trial
This is an interventional treatment trial for Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) focused on measuring Myelodysplastic Syndromes, Anemia, Hemoglobin (Hb), Low Risk Myelodysplastic Syndrome, Low Risk MDS
Eligibility Criteria
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 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 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.
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Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Roxadustat
Placebo
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.
Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks.