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

NCT04477850 | Completed Phase 2

• 2 other identifiers: ACE-536-MDS-004U1111-1251-9249
• Study Type: interventional
• Target: 30
• Locations: 2 countries
• Sites: 25

Brief Summary

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.

Conditions

Myelodysplastic Syndromes

Keywords

Myelodysplastic Syndromes; MDS; ACE-536; Anemia; Luspatercept

Outcome Measures

Primary Outcomes (1)

• Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks

  Week 1 through Week 24

Secondary Outcomes (18)

• RBC-TI ≥ 12 weeks

  Week 1 through Week 24

• Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline

  Week 9 through Week 24

• Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG)

  Week 1 through Week 24

• Mean hemoglobin increase ≥ 1.0 g/dL

  Week 1 through Week 24

• Duration of RBC-TI

  Week 1 through Week 24

Study Arms (1)

Luspatercept Administration

EXPERIMENTAL

Drug: Luspatercept

Interventions

Luspatercept DRUG

Specified dose on specified days

Also known as: ACE-536

Luspatercept Administration

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Celgene: lead

Study Sites (25)

Local Institution - 100

Beijing, 100730, China

Location

Local Institution - 107

Chengdu, Sichuan, 610041, China

Location

Local Institution - 105

Guangzhou, 510060, China

Location

Local Institution - 103

Guangzhou, 510080, China

Location

Local Institution - 109

Guangzhou, 510515, China

Location

Local Institution - 102

Hangzhou, 310006, China

Location

Local Institution - 112

Nanchang, 330006, China

Location

Local Institution - 108

Nanjing, 210029, China

Location

Local Institution - 114

Shanghai, 0, China

Location

Local Institution - 101

Shanghai, 200233, China

Location

Local Institution - 104

Suzhu, 215006, China

Location

Local Institution - 106

Tianjin, 300020, China

Location

Local Institution - 111

Wenzhou, 325000, China

Location

Local Institution - 110

Wuhan, 430022, China

Location

Local Institution - 209

Matsuyama, Ehime, 790-8524, Japan

Location

Local Institution - 203

Nagasaki, Nagasaki, 8528511, Japan

Location

Local Institution - 210

Sayama, Osaka, 5898511, Japan

Location

Local Institution - UNK11

Fukuoka, 810-8563, Japan

Location

Local Institution - 206

Kamogawa, 296-8602, Japan

Location

Local Institution - 201

Mibu-Machi, 321-0293, Japan

Location

Local Institution - 205

Osaka, 545-8585, Japan

Location

Local Institution - 208

Ōgaki, 503-8502, Japan

Location

Local Institution - 204

Sagamihara, 252-0375, Japan

Location

Local Institution - 207

Sendai, 980-8574, Japan

Location

Local Institution - 202

Shinagawa-ku, Tokyo, 141-8625, Japan

Location

Related Publications (1)

• Chang C, Suzuki T, Liang Y, Tong H, Usuki K, Liu Q, Wu Y, Fujisaki T, Han B, Huang R, Morita Y, Miao M, Nakashima Y, Tian YO, Pu J, Aggarwal D, Pozharskaya V, Shi W, Xiao Z, Mitani K. Safety and efficacy of luspatercept in treating anemia associated with myelodysplastic syndrome with ring sideroblasts in Asian patients who require red blood cell transfusions: a phase II bridging study. Ther Adv Hematol. 2025 Feb 20;16:20406207251321715. doi: 10.1177/20406207251321715. eCollection 2025.

  PMID: 39991012 DERIVED

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2020
• First Posted: July 20, 2020
• Study Start: November 30, 2020
• Primary Completion: September 29, 2023
• Study Completion: April 8, 2026
• Last Updated: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

CN (14); JP (11)