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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

Primary Purpose

Myelodysplastic Syndromes

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Luspatercept

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring Myelodysplastic Syndromes, MDS, ACE-536, Anemia, Luspatercept

Eligibility Criteria

20 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Luspatercept Administration

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

RBC-TI ≥ 12 weeks

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

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

Mean hemoglobin increase ≥ 1.0 g/dL

Duration of RBC-TI

Mean decrease in serum ferritin compared to baseline

Mean decrease in iron chelation therapy (ICT) use compared to baseline

Time to RBC-TI

Progression to acute myeloid leukemia (AML)

Overall survival (OS)

Incidence of type of adverse events (AEs)

Incidence of frequency of AEs

Incidence of severity of AEs

Incidence of seriousness of AEs

Incidence of relationship of AEs to study treatment

Pharmacokinetics - Area under the curve (AUC)

Pharmacokinetics - Maximum plasma concentration of the drug (Cmax)

Frequency of Anti-drug antibodies (ADA)

Full Information

NCT ID

NCT04477850

First Posted

June 23, 2020

Last Updated

October 10, 2023

Sponsor

Celgene

1. Study Identification

Unique Protocol Identification Number

NCT04477850

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

November 30, 2020 (Actual)

Primary Completion Date

September 29, 2023 (Actual)

Study Completion Date

February 23, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Celgene

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplastic Syndromes

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

30 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Luspatercept Administration

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Luspatercept

Other Intervention Name(s)

ACE-536

Intervention Description

Specified dose on specified days

Primary Outcome Measure Information:

Title

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

Time Frame

Week 1 through Week 24

Secondary Outcome Measure Information:

Title

RBC-TI ≥ 12 weeks

Time Frame

Week 1 through Week 24

Title

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

Time Frame

Week 9 through Week 24

Title

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

Time Frame

Week 1 through Week 24

Title

Mean hemoglobin increase ≥ 1.0 g/dL

Time Frame

Week 1 through Week 24

Title

Duration of RBC-TI

Time Frame

Week 1 through Week 24

Title

Mean decrease in serum ferritin compared to baseline

Time Frame

Week 9 through Week 24

Title

Mean decrease in iron chelation therapy (ICT) use compared to baseline

Time Frame

Week 9 through Week 24

Title

Time to RBC-TI

Time Frame

Week 1 through Week 24

Title

Progression to acute myeloid leukemia (AML)

Time Frame

Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose

Title

Overall survival (OS)

Time Frame

Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose

Title

Incidence of type of adverse events (AEs)

Time Frame