Luspatercept

Generic Name: Luspatercept

Brand Names: Reblozyl

Drug Type: Biotech

CAS Number: 1373715-00-4

Unique Ingredient Identifier: AQK7UBA1LS

Background: Luspatercept is a recombinant fusion protein comprised of a modified extracellular domain of activin receptor type IIB fused to the FC domain of human IgG1. It was first approved for use in the United States in November 2019 under the brand name Reblozyl® for the treatment of anemia in patients with beta thalassemia who require regular blood transfusions. Luspatercept is novel in that it ameliorates anemia via action on late-stage erythropoiesis, in contrast to typical erythropoiesis-stimulating agents (ESAs), such as darbepoetin alfa and epoetin alfa, which act only on early-stage erythropoiesis. Luspatercept's novel mechanism of action, then, is uniquely suited for the treatment of conditions in which late-stage erythropoiesis is defective, such as beta thalassemia and other myelodysplastic diseases.

Indication: Luspatercept is indicated for the treatment of:

Associated Conditions: Anemia

Clinical Trials

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions

Phase 3

Active, not recruiting

Conditions: Myeloproliferative Disorders
Myelofibrosis
Primary Myelofibrosis
Post-Polycythemia Vera Myelofibrosis
Anemia

Interventions: Drug: ACE-536
Other: Placebo

First Posted Date: 2021-01-22

Last Posted Date: 2025-03-14

Lead Sponsor: Celgene

Target Recruit Count: 309

Registration Number: NCT04717414

Locations: 🇺🇸
University Of California Los Angeles, Los Angeles, California, United States
🇺🇸
BRCR Medical Center Inc., Plantation, Florida, United States
🇺🇸
Local Institution - 112, Chicago, Illinois, United States

and more 205 locations

Luspatercept and Lenalidomide (L2) in Lower-risk, Non-del(5q) MDS Patients

Phase 1

Recruiting

Conditions: Myelodysplastic Syndromes

Interventions: Drug: Lenalidomide
Drug: Luspatercept

First Posted Date: 2020-09-04

Last Posted Date: 2025-01-29

Lead Sponsor: Mikkael Sekeres, MD

Target Recruit Count: 50

Registration Number: NCT04539236

Locations: 🇺🇸
University of Texas Southwestern Medical Center, Dallas, Texas, United States
🇺🇸
University of Miami, Miami, Florida, United States
🇺🇸
Moffitt Cancer Center, Tampa, Florida, United States

and more 4 locations

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 2

Active, not recruiting

Conditions: Myelodysplastic Syndromes

Interventions: Drug: Luspatercept

First Posted Date: 2020-07-20

Last Posted Date: 2023-10-12

Lead Sponsor: Celgene

Target Recruit Count: 30

Registration Number: NCT04477850

Locations: 🇯🇵
Local Institution - 203, Nagasaki-shi, Nagasaki, Japan
🇨🇳
Local Institution - 105, Guangzhou, China
🇨🇳
Local Institution - 103, Guangzhou, China

and more 21 locations

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Phase 2

Recruiting

Conditions: Beta-Thalassemia

Interventions: Drug: ACE-536

First Posted Date: 2019-10-29

Last Posted Date: 2025-05-18

Lead Sponsor: Celgene

Target Recruit Count: 99

Registration Number: NCT04143724

Locations: 🇨🇳
West China Hospital - Sichuan University, Chengdu, Sichuan, China
🇺🇸
Local Institution - 601, Los Angeles, California, United States
🇺🇸
New York Presbyterian Hospital, New York, New York, United States

and more 22 locations

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Phase 3

Recruiting

Conditions: Myelodysplastic Syndromes (MDS)
Beta-thalassemia
Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

Interventions: Drug: Luspatercept

First Posted Date: 2019-08-21

Last Posted Date: 2025-05-09

Lead Sponsor: Celgene

Target Recruit Count: 665

Registration Number: NCT04064060

Locations: 🇩🇪
Local Institution - 349, Leipzig, Germany
🇩🇪
Local Institution - 340, Mainz, Germany
🇮🇱
Local Institution - 424, Jerusalem, Israel

and more 140 locations

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

Phase 2

Completed

Conditions: Myelodysplastic Syndromes

Interventions: Drug: Luspatercept

First Posted Date: 2019-04-03

Last Posted Date: 2024-10-17

Lead Sponsor: Celgene

Target Recruit Count: 21

Registration Number: NCT03900715

Locations: 🇯🇵
Local Institution - 344, Nagasaki-shi, Nagasaki, Japan
🇯🇵
Local Institution - 331, Kamogawa, Japan
🇯🇵
Local Institution - 348, Kitakyushu, Japan

and more 16 locations

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve

Phase 3

Active, not recruiting

Conditions: Myelodysplastic Syndromes

Interventions: Drug: Luspatercept
Drug: Epoetin alfa

First Posted Date: 2018-09-24

Last Posted Date: 2024-11-20

Lead Sponsor: Celgene

Target Recruit Count: 363

Registration Number: NCT03682536

Locations: 🇺🇸
Local Institution - 114, Salt Lake City, Utah, United States
🇺🇸
Local Institution - 107, Berkeley, California, United States
🇺🇸
Local Institution - 115, San Diego, California, United States

and more 223 locations

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

Phase 2

Completed

Conditions: Thalassemia

Interventions: Other: Placebo
Drug: Luspatercept
Other: Best Supportive Care (BSC)

First Posted Date: 2017-11-17

Last Posted Date: 2023-12-20

Lead Sponsor: Celgene

Target Recruit Count: 145

Registration Number: NCT03342404

Locations: 🇬🇷
Local Institution - 102, Athens, Greece
🇺🇸
Children's Hospital and Research Center at Oakland, Oakland, California, United States
🇺🇸
Children's Hospital of Los Angeles, Los Angeles, California, United States

and more 13 locations

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence

Phase 2

Completed

Conditions: Anemia
Primary Myelofibrosis

Interventions: Drug: Luspatercept

First Posted Date: 2017-06-21

Last Posted Date: 2023-08-24

Lead Sponsor: Celgene

Target Recruit Count: 95

Registration Number: NCT03194542

Locations: 🇺🇸
Local Institution - 109, Cleveland, Ohio, United States
🇺🇸
Local Institution - 100, Houston, Texas, United States
🇺🇸
MD Anderson Cancer Center The University of Texas, Houston, Texas, United States

and more 44 locations

A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Phase 3

Completed

Conditions: Myelodysplastic Syndromes

Interventions: Other: Placebo
Drug: Luspatercept

First Posted Date: 2015-12-15

Last Posted Date: 2021-12-17

Lead Sponsor: Celgene

Target Recruit Count: 229

Registration Number: NCT02631070

Locations: 🇺🇸
Yale University School of Medicine, New Haven, Connecticut, United States
🇺🇸
H Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, United States
🇩🇪
Universitatsklinikum Bonn, Bonn, Germany

and more 71 locations

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