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

Myelodysplastic Syndromes Clinical Trial

Official title:

A Phase 2, Multicenter, Single-Arm Bridging Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes(MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04477850
• Other study ID #: ACE-536-MDS-004
• Secondary ID: U1111-1251-9249
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: November 30, 2020
• Est. completion date: February 23, 2026

Study information

• Verified date: October 2023
• Source: Celgene
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: February 23, 2026
• Est. primary completion date: September 29, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Drug:
• Luspatercept
Specified dose on specified days

Locations

Country	Name	City	State
China	Local Institution - 100	Beijing
China	Local Institution - 107	Chengdu, Sichuan
China	Local Institution - 103	Guangzhou
China	Local Institution - 105	Guangzhou
China	Local Institution - 109	Guangzhou
China	Local Institution - 102	Hangzhou City
China	Local Institution - 112	Nanchang
China	Local Institution - 108	Nanjing
China	Local Institution - 101	Shanghai
China	Local Institution - 114	Shanghai
China	Local Institution - 104	Suzhu
China	Local Institution - 106	Tianjin
China	Local Institution - 111	Wenzhou
China	Local Institution - 110	Wuhan
Japan	Local Institution - 206	Kamogawa
Japan	Local Institution - 209	Matsuyama	Ehime
Japan	Local Institution - 201	Mibu-Machi
Japan	Local Institution - 203	Nagasaki-shi	Nagasaki
Japan	Local Institution - 208	Ogaki
Japan	Local Institution - 205	Osaka
Japan	Local Institution - 210	Osakasayama	Osaka
Japan	Local Institution - 204	Sagamihara
Japan	Local Institution - 207	Sendai
Japan	Local Institution - 202	Shinagawa-ku, Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Celgene

Countries where clinical trial is conducted

China,  Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Red Blood Cell Transfusion Independence (RBC-TI) = 8 weeks		Week 1 through Week 24
Secondary	RBC-TI = 12 weeks		Week 1 through Week 24
Secondary	Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline		Week 9 through Week 24
Secondary	Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG)		Week 1 through Week 24
Secondary	Mean hemoglobin increase = 1.0 g/dL		Week 1 through Week 24
Secondary	Duration of RBC-TI		Week 1 through Week 24
Secondary	Mean decrease in serum ferritin compared to baseline		Week 9 through Week 24
Secondary	Mean decrease in iron chelation therapy (ICT) use compared to baseline		Week 9 through Week 24
Secondary	Time to RBC-TI		Week 1 through Week 24
Secondary	Progression to acute myeloid leukemia (AML)		Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Secondary	Overall survival (OS)		Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Secondary	Incidence of type of adverse events (AEs)		Screening through 42 days post last dose
Secondary	Incidence of frequency of AEs		Screening through 42 days post last dose
Secondary	Incidence of severity of AEs		Screening through 42 days post last dose
Secondary	Incidence of seriousness of AEs		Screening through 42 days post last dose
Secondary	Incidence of relationship of AEs to study treatment		Screening through 42 days post last dose
Secondary	Pharmacokinetics - Area under the curve (AUC)		Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Secondary	Pharmacokinetics - Maximum plasma concentration of the drug (Cmax)		Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Secondary	Frequency of Anti-drug antibodies (ADA)		Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose

External Links

•   BMS Clinical Trial Information
•   BMS Clinical Trial Patient Recruiting