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Myelodysplastic SyndromesPhase 2NCT05732961

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

A Phase 2 study testing Luspatercept in people with Myelodysplastic Syndromes and Myeloproliferative Neoplasm, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Luspatercept
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
21 Feb 2023to Sep 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 26Show fewer
  • 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  • Refractory to prior ESA treatment - non-response or response that is no longer maintained.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
  • Require RBC transfusions
  • Average of ≥ 2 units/8 weeks of pRBCs confirmed for a minimum of 16 weeks immediately preceding registration
  • Applies to on treatment subjects only - females of childbearing potential (FCBP) defined as a sexually mature woman who:
  • has achieved menarche at some point,
  • has not undergone a hysterectomy or bilateral oophorectomy, or
  • has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must:
  • Have two negative pregnancy tests 48 hours apart as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence* from heterosexual contact.
  • Either commit to true abstinence*from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting
  • investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy
  • Applies to on treatment subjects only - Male subjects must:
  • Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy. * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception).

Who cannot join

  • 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
Show all 10Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Moffitt Cancer Center

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase 2, Single Arm Study of Luspatercept for the Treatment of Anemia in Lower Risk Myelodysplastic Syndromes (MDS) or Non-Proliferative Myelodysplastic Syndromes/ Myeloproliferative Neoplasms (MDS/MPN)

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.

Being tested
Luspatercept
Keywords
MDS, MPN, Non-Proliferative MDS, Non-Proliferative MPN
Medical subject headings
Myelodysplastic Syndromes, Myeloproliferative Disorders, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LuspaterceptDrug

    Also called ACE-536

    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)

Study groups in NCT05732961
Study groupRoleWhat they receive
Participants with gene mutations other than SF3B1ExperimentalParticipants with lower risk MDS or non-proliferative MDS/MPN with somatic splicing gene mutations other than SF3B1
Receives: Drug: Luspatercept
Participants with SF3B1 mutationExperimentalParticipants with lower risk MDS or non-proliferative MDS/MPN with SF3B1 mutation who had received hypomethylating agents and or lenalidomide.
Receives: Drug: Luspatercept

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. RBC Transfusion Independence

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

    Measured: From start of treatment to up to 18 months

Secondary outcomes (4)

  1. Incidence of treatment related adverse events

    To determine the number of participants with treatment related AEs using CTCAE v5

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

  2. Hematological Improvement

    Hematological improvement as defined by using IWG 2006 MDS response criteria

    Measured: From start of treatment to up to 18 months

  3. Duration of Response

    The duration of response is measured from the time measurement criteria are met for RBC TI or HI by IWG 2006 criteria until the first date of loss of response or progressive disease is objectively documented.

    Measured: From start of treatment to up to 18 months

  4. ASC specks changes with response

    ASC specks as biomarker of response, investigators will compare mean baseline percentage of ASC specks among responders and non-responders (t-test) and use paired t-test to compare change in mean percentage of ASC specks with treatment among responders and non-responders

    Measured: End of treatment, up to 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Feb 2023
  2. Study start21 Feb 2023
  3. Primary completion26 Mar 2026
  4. Study completionSep 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05732961
Sponsor's protocol ID
MCC-21405
Status last verified
Apr 2026
First submitted
8 Feb 2023
Last updated
5 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Luspatercept in people with Myelodysplastic Syndromes and Myeloproliferative Neoplasm, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 26 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

RBC Transfusion Independence (time frame: From start of treatment to up to 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05732961.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2026.

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