Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)

FibroGen

Status and phase: Completed; Phase 3; Phase 2

Conditions: Anemia; Myelodysplastic Syndrome

Treatments: Drug: FG-4592; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03303066

FGCL-4592-813

Details and patient eligibility

About

The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in Chinese participants with lower risk MDS.

Full description

This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week follow-up period.

Enrollment: 43 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts (documented within 12 weeks prior to Day 1)
• Screening Hb <10 g/dL and ≥6g/dL
• Transfusion independent defined as no red blood cell transfusions within 12 weeks of Day 1
• Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1)
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2

Key Exclusion Criteria:

• Diagnosis of secondary MDS
• Significant myelofibrosis (>2+fibrosis)
• Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to Day 1
• Baseline erythropoietin level of >400 units (U)/liter (L)
• Clinically significant anemia due to non-MDS etiologies

Note: Other inclusion and exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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43 participants in 3 patient groups, including a placebo group

Phase 2 Part: FG-4592

Experimental group

Description:

Fixed starting doses (different doses for lower body weight \& higher body weight) of FG-4592 administered orally 3 times a week (TIW) for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.

Treatment:

Drug: FG-4592

Phase 3 Part: FG-4592

Experimental group

Description:

Fixed starting doses (different doses for lower body weight \& higher body weight) of FG-4592 administered orally TIW for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.

Treatment:

Drug: FG-4592

Phase 3 Part: Placebo

Placebo Comparator group

Description:

Placebo (matching to FG-4592) administered orally TIW for up to 26 weeks.

Treatment:

Drug: Placebo