Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion Burden

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Brief Summary

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) Description: Up to 24 patients overall will be enrolled in sequential dose level cohorts prior to start of the double-blind portion of the study. All patients will receive roxadustat in an open-label manner. The first 8 will receive a starting dose of 1.5 mg/kg. The next 8 will receive a starting dose of 2.0 mg/kg. The last 8 will receive a starting dose of 2.5 mg/kg. The initial open-label portion with up to of 24 patients who receive roxadustat may remain in the study up to 52 weeks of treatment based on investigator discretion, adequate safety, and impression of benefit. These patients will undergo all procedures as will the randomized patients with the exception of not completing the Patient Reported Outcomes (PRO) assessments. The double-blind portion of the study will open once the dose escalation portion completes. Here's the for breakdown of the enrollment for the entire study: *open-label portion: 24 patients *double-blind portion: 160 patients *A total of 184 patients will be enrolled in this study. We plan on enrolling 40 patients at UCLA with a goal of 10 patients in the open-label portion and 30 patients in the double-blind portion.

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase III

Eligibility

Gender

All

Healthy Volunteers

Minimum Age

18 Years

Maximum Age

N/A

Adult subjects diagnosed with anemia with lower risk Myelodysplastic Syndrome (MDS) with Low Red Blood Cell (RBC) Transfusion Burden (LTB). For more information about the eligibility criteria for this trial, refer to the Health Professional version.

Key Inclusion Criteria:

Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts. There is no minimum time from diagnosis except to allow for proper IPSS-R classification to be made, and to show transfusion dependence.
RBC transfusion of either 2-4 pRBC units over the 8 weeks prior to randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to randomization
No restriction on prior use of ESAs, except no ESA use within 8 weeks prior to registration/randomization
Pre-transfusion hemoglobin of <= 10 g/dL,
ECOG of 0-2 at screen
History of cured malignancy with no evidence of recurrence for a least 3 years are eligible

Key Exclusion Criteria:

Diagnosis of secondary MDS
Significant myelofibrosis (>2+fibrosis)
MDS associated with 5q(del) abnormality
Screen serum erythropoietin level > 400 mIU/mL (Double Blind); for Open- Label High Erythropoietin patients serum erythropoietin level must be >400 mIU/mL
Clinically significant anemia due to non-MDS etiologies

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