A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Phase 2Active Not RecruitingNCT04477850

Celgene30 enrolled

Overview

The purpose of this study is to evaluate the efficacy and safety of luspatercept (ACE-536) for the treatment of anemia due to Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) in Chinese and Japanese participants with ring sideroblasts who require Red Blood Cells (RBC) transfusions.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Syndromes

Interventions

LuspaterceptDRUG

Specified dose on specified days

Eligibility

Sex: ALLMin age: 20 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Refractory or intolerant to, or ineligible for, prior Erythropoiesis stimulating agent (ESA) treatment as defined by any one of the following: Refractory to prior ESA treatment, Intolerant to prior ESA treatment, or ESA ineligible. * previously treated with an ESA or granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, both agents must have been discontinued ≥ 4 weeks prior to date of luspatercept treatment * Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 Exclusion Criteria: * Prior therapy with disease modifying agents for underlying MDS disease * Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding * Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN) Other protocol-defined inclusion/exclusion criteria apply

Locations (24)

Local Institution - 100

Beijing, China

Local Institution - 107

Chengdu, Sichuan, China

Local Institution - 105

Guangzhou, China

Local Institution - 103

Guangzhou, China

Local Institution - 109

Guangzhou, China

Local Institution - 102

Hangzhou, China

Local Institution - 112

Nanchang, China

Local Institution - 108

Nanjing, China

Local Institution - 114

Shanghai, China

Local Institution - 101

Shanghai, China

...and 14 more locations