Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms

NCT05732961 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MCC-21405
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to see if participants with anemia due to their type of MDS or MDS/MPN will experience a more decreased need for regular blood transfusions if they take luspatercept plus best supportive care, and what effect, good and/or bad, luspatercept has on them and their anemia due to MDS or MDS/MPN. The safety and tolerability of luspatercept will also be evaluated in this study.

Conditions

Myeloproliferative Neoplasm; Anemia; Myelodysplastic Syndromes

Keywords

MDS; MPN; Non-Proliferative MDS; Non-Proliferative MPN

Outcome Measures

Primary Outcomes (1)

• RBC Transfusion Independence

  RBC transfusion independence (RBC-TI) as defined by IWG 2006 MDS response criteria

  From start of treatment to up to 18 months

Secondary Outcomes (4)

• Incidence of treatment related adverse events

  From start of treatment to 30 days after the last day of treatment, up to 19 months

• Hematological Improvement

  From start of treatment to up to 18 months

• Duration of Response

  From start of treatment to up to 18 months

• ASC specks changes with response

  End of treatment, up to 18 months

Study Arms (2)

Participants with gene mutations other than SF3B1

EXPERIMENTAL

Participants with lower risk MDS or non-proliferative MDS/MPN with somatic splicing gene mutations other than SF3B1

Drug: Luspatercept

Participants with SF3B1 mutation

EXPERIMENTAL

Participants with lower risk MDS or non-proliferative MDS/MPN with SF3B1 mutation who had received hypomethylating agents and or lenalidomide.

Drug: Luspatercept

Interventions

Luspatercept DRUG

Participants will be treated with Luspatercept, with a starting dose of 1.0 mg/kg subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle)

Also known as: ACE-536

Participants with SF3B1 mutation; Participants with gene mutations other than SF3B1

Eligibility Criteria

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

You may qualify if:

• Participant is ≥18 years at the time of signing the informed consent form
• Participant is willing and able to adhere to the study visit schedule and other protocol requirements
• Documented diagnosis of MDS or non-proliferative MDS/MPN (WBC \< 13,000 U/L)
• According to WHO 2016 classification
• Meets IPSS-R classification of very low, low, or intermediate risk disease
• Documented acquired splicing gene mutation
• Cohort 1: detectable splicing mutation other than SF3B1: (SRSF2, U2AF1, ZRSR2)
• Cohort 2: SF3B1 mutation with prior treatment with hypomethylating agent and or lenalidomide
• \<5% blasts in bone marrow
• Refractory, intolerant to, or ineligible for, prior ESA treatment, as defined by any one of the following:
• Refractory to prior ESA treatment - non-response or response that is no longer maintained. ESA regimen must have been either:
• rHu EPO ≥ 40,000 IU/wk for at least 8 doses or equivalent Or darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent
• Intolerant to prior ESA treatment - discontinuation of prior ESA-containing regimen, at any time after introduction due to intolerance or AE
• ESA ineligible - Low chance of response to ESA based on endogenous serum EPO \> 200 U/L for subjects not previously treated with ESAs
• Discontinuation of ESAs, G-CSF, GM-CSF ≥ 4 weeks prior to start of study treatment

You may not qualify if:

• Prior allogeneic or autologous stem cell transplant
• MDS associated with del 5q cytogenetic abnormality if no prior lenalidomide treatment
• Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment
• ANC \< 500/μL (0.5 x 109/L)
• Platelet count ˂50,000/μL (50 x 109/L)
• Active other malignancies
• Severe renal impairment (eGFR \< 30 mL/min/1.73 m2)
• ALT or AST ≥ 3 × ULN
• Prior treatment with Luspatercept or Sotatercept
• Pregnant or breastfeeding females

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Myeloproliferative Disorders; Anemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Rami Komrokji, MD

  Moffitt Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: February 8, 2023
• First Posted: February 17, 2023
• Study Start: February 21, 2023
• Primary Completion: March 26, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: May 5, 2026

Record last verified: 2026-04

Locations

US (1)