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CCUS Clonal Cytopenia of Undetermined SignificancePhase 2NCT06788691

Luspatercept for Clonal Cytopenias of Uncertain Significance

A Phase 2 study testing Luspatercept in people with CCUS Clonal Cytopenia of Undetermined Significance and Anemia, run by Weill Medical College of Cornell University at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). 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
50 participants
Where
1 siteUnited States
Study dates
25 Mar 2025to Feb 2028
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

  • Male or female ≥ 18 years of age.
  • Documentation of a CCUS diagnosis.
  • Clonal cytopenia of undetermined significance (CCUS) is defined as clonal hematopoiesis of indeterminate potential (CHIP) detected in the presence of one or more persistent cytopenias that are otherwise unexplained by hematologic or non-hematologic conditions and that do not meet diagnostic criteria for defined myeloid neoplasms. Cytopenia definitions for diagnosis of CCUS include Hb <13 g/dL in males and <12 g/dL in females for anemia, absolute neutrophil count <1.8 ×109/L for leukopenia, and platelets <150 × 109/L for thrombocytopenia.
  • Patients should harbor somatic mutations of myeloid malignancy-associated genes detected in the blood or bone marrow at a variant allele fraction (VAF) of ≥ 2% (≥4% for X-linked gene mutations in males
  • Clinically significant cytopenias demonstrated in two separate lab draws and defined as cytopenia in any one of the following:
  • Anemia: Transfusion dependent (LTD or HTD for Hb < 9 g/dL based on IWG 2018 criteria). Exception for higher threshold up to 10g/dL for documented moderate or severe angina pectoris, cardiac or pulmonary insufficiency, or ischemic neurologic diseases (per IWG 2018 consensus recommendation).
Show all 22Show fewer
  • Anemia NTD: symptomatic NTD CCUS with Hb <10 g/dl, symptomatic defined as moderate or worse on ≥ 1 Patient Global Impression of Severity (PGI-S) item (fatigue, shortness of breath, weakness, or dizziness)
  • Thrombocytopenia: platelet count less than 30,000 /microL or < 50,000/microL with documented bleeding events or high risk for bleeding, for example on blood thinners or drugs that inhibit platelet function for other comorbidities.
  • Neutropenia: Neutropenia below 750/microl are included in the study. For subjects with neutropenia between 750-1000/microl, subjects should have neutropenia AND a history of serious infection(s).
  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2
  • Adequate organ function as defined by:
  • Direct bilirubin < 3 x ULN. Indirect hyperbilirubinemia from hemolysis or Gilberts disease are not considered as impaired.
  • Estimated Creatinine clearance or GFR >30 ml/min by institutional standards (for example MDRD or Cockcroft Gault or measured by 24 hour urine clearance).
  • ALT and AST < 3 x ULN
  • Females of childbearing potential (FCBP), defined as a sexually mature woman who: 1) has achieved menarche at some point, 2) not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy or amenorrhea due to other medical reasons does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months), must:
  • Have two negative pregnancy tests (serum or urine) as verified by the investigator prior to starting study therapy (unless the screening pregnancy test was done within 72 hours of W1D1). She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment.
  • Either commit to true abstinence1 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 contraception2 without interruption, 5 weeks prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks after discontinuation of study therapy.
  • Male subjects must:
  • Practice true abstinence1(which must be reviewed prior to each IP administration or on a monthly basis [e.g., in the event of dose delays]) or agree to use a condom (latex or non-latex, but not made out of natural [animal] membrane) 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 12 weeks following investigational product discontinuation, even if he has undergone a successful vasectomy.
  • Contraception
  • True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception].
  • Highly effective contraception is defined in this protocol as the following (information will also appear in the ICF): Hormonal contraception (for example, birth control pills, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation (tying your tubes); or a partner with a successful vasectomy.

Who cannot join

  • Concurrent malignancy requiring active concurrent systemic chemotherapy. Hormonal therapy for malignancy and targeted radiation is allowed. If patients after enrollment, have a clinical need for chemotherapy after achieving response on treatment, subjects deriving clinical benefit can be continued on study after discussion with study PI.
  • Diagnosis of MDS, AML, MPN or any other myeloid malignancy in the patient's lifetime
  • Active uncontrolled infection that in the investigators opinion will affect study procedures and/or results
  • Active uncontrolled hypertension not responding to blood pressure lowering medications which in the investigator's opinion will be harmful for the patient.
  • Use of ESA or growth factors within four weeks prior to the start of the study
  • Known risk factors for thromboembolism (splenectomy, concomitant use of hormone replacement therapy or recent uncontrolled pulmonary embolism or DVT in the last 6 months). Subjects adequately controlled on anticoagulation are permitted.
  • Pregnant or nursing women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study treatment and for up to 130 days after last dose of study drug. Basic contraception methods are defined in Section 4.4.
  • Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least six weeks prior to first dose of study drug. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of childbearing potential. If local regulations deviate from the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the Informed Consent Form (ICF).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Pinkal Desai, MD contact

646-962-2700

pid9006@med.cornell.edu

Ameenah Sukkur, BA contact

646-962-4580

ams4015@med.cornell.edu

United States1 site, 1 recruiting

  • Weill Cornell Medical College

    New York, New York, 10021

    Pinkal Desai, MD, 646-962-2700, pid9006@med.cornell.edu

    Ameenah Sukkur, BA, 646-962-4580, ams4015@med.cornell.edu

    Recruiting

Other trials in United States

About this study

Official title: Efficacy of Luspatercept In Clonal Cytopenias of Uncertain Significance

The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). The main questions the study seeks to answer include:

  • How many patients experience improvements in their low blood counts (red cells, platelets, or white cells) within 24 weeks, based on specific criteria for blood conditions like myelodysplastic syndromes (MDS)?
  • How long these improvements last before the condition worsens or changes.
  • The percentage of participants showing improvements at 12, 24, and 48 weeks.
  • How long it takes for the condition to progress to more severe diseases like myeloid disorders.
  • How long red blood cell responses last and how quickly these responses are seen.
  • The average change in hemoglobin levels over 24 weeks.
  • How many patients need blood transfusions during the study and how soon transfusions are required.
  • Changes in participants' well-being and energy levels based on a standardized questionnaire.
  • Monitoring for any side effects, including progression to MDS or leukemia, heart-related issues, or sudden increases in hemoglobin.
Read more

Participants will:

  • Receive luspatercept as an injection every three weeks.
  • Visit the clinic every three weeks for treatment and monitoring.
Being tested
Luspatercept
Medical subject headings
Anemia, Leukopenia, Thrombocytopenia, Neutropenia

What participants receive

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

  • LuspaterceptDrug

    Administered at 1 mg/kg once every 3 weeks

Study groups in NCT06788691
Study groupRoleWhat they receive
LuspaterceptExperimentalLuspatercept administered at 1 mg/kg IV once every 3 weeks
Receives: Drug: Luspatercept

What is being measured

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

  1. Number of patients achieving cytopenia responses as defined by HI-E, HI-P, HI-N, mHI-E responses

    Adapted from International Working Group (IWG) 2018 response definition for Myelodysplastic Syndromes

    Measured: 24 Weeks

Secondary outcomes (25)

  1. Duration of cytopenia response

    The duration of cytopenia response, defined as the time from the first documented achievement of cytopenia response to the loss of response or disease progression.

    Measured: 24 Weeks

  2. Percentage of Participants Achieving Hematologic Response

    Measured: 12 Weeks

  3. Percentage of Participants Achieving Hematologic Response

    Measured: 24 Weeks

  4. Percentage of Participants Achieving Hematologic Response

    Measured: 48 Weeks

  5. Time to Transformation to Myeloid Disease

    Time to transformation to myeloid disease as evidenced by bone marrow morphology findings.

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  6. Duration of modified hematologic improvement-erythroid (mHI-E)

    Duration of modified hematologic improvement-erythroid (HI-E) per IWG 2006

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  7. Time to mHI-E

    Time to hematologic improvement-erythroid (mHI-E) per IWG 2006

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  8. Mean Change Hemoglobin (Hb)

    Measured: Baseline, 24 Weeks

  9. Number of Patients Converting to Transfusion Dependence (TD)

    Number of patients who convert to transfusion dependence (≥ 3 units/16 weeks per IWG 2018 criteria) at 12 weeks, provided they were not transfusion-dependent at study entry.

    Measured: 12 Weeks

  10. Number of Patients Converting to Transfusion Dependence (TD)

    Number of patients who convert to transfusion dependence (≥ 3 units/16 weeks per IWG 2018 criteria) at 24 weeks, provided they were not transfusion-dependent at study entry.

    Measured: 24 Weeks

  11. Number of Patients Converting to Transfusion Dependence (TD)

    Number of patients who convert to transfusion dependence (≥ 3 units/16 weeks per IWG 2018 criteria) at 48 weeks, provided they were not transfusion-dependent at study entry.

    Measured: 48 Weeks

  12. Time to the first red blood cell transfusion

    Measured: 24 Weeks

  13. Mean Change in FACT-An Total Scores

    Functional Assessment of Cancer Therapy-Anemia (FACT-An) total scores. The FACT-An (Functional Assessment of Chronic Illness Therapy - Anemia) questionnaire is a validated tool designed to evaluate the quality of life in patients with anemia, especially those receiving treatment for cancer-related anemia. The total FACT-An score ranges from 0 to 188.

    Measured: Baseline, 24 Weeks

  14. Mean Change in FACT-An Subscale Scores

    Functional Assessment of Cancer Therapy-Anemia (FACT-An) subscale scores. The FACT-An (Functional Assessment of Chronic Illness Therapy - Anemia) questionnaire is a validated tool designed to evaluate the quality of life in patients with anemia, especially those receiving treatment for cancer-related anemia. The questionnaire consists of five subscales, with score ranges varying by subscale: 0-28, 0-24, or 0-80.

    Measured: Baseline, 24 Weeks

  15. Safety as assessed by number of subjects with MDS/AML progression

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  16. Safety as assessed by number of cardiovascular events

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  17. Safety as assessed by number of subjects with rapid hemoglobin rise

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  18. Number of participants achieving Red Blood Cell Transfusion Independence (RBC-TI) for at least 24 consecutive weeks

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  19. Number of participants achieving Hematologic Improvement-Erythroid (HI-E) for at least 24 consecutive week as per IWG 2006 criteria.

    Measured: Week 4 (Cycle 1 Week 1) to 24 Weeks

  20. Duration of RBC-TI greater than 16 Weeks

    Duration of red blood cell transfusion independence (RBC-TI) for greater than 16 weeks

    Measured: Week 1 to Week 24

  21. Number of participants achieving RBC-TI

    Number of participants achieving RBC-TI over 16 weeks along with a mean hemoglobin increase of >1.5 g/dL over the same time period.

    Measured: 16 Weeks

  22. Change in cardiac biomarkers over the treatment period

    Measured: Baseline, 24 Weeks

  23. Mean Change Health-related quality of life (HRQoL) Assessment

    The Functional Assessment of Cancer Therapy - Anemia (FACT-An) is the Health-Related Quality of Life (HRQoL) tool used in this study. The lowest possible score is 0, which reflects the worst possible quality of life, indicating severe impairments in overall well-being and significant anemia-related symptoms. The highest possible score is 188, representing the best possible quality of life, with minimal or no symptoms and optimal functioning across all domains, including anemia-related concerns.

    Measured: 12 Weeks

  24. Mean Change Health-related quality of life (HRQoL) Assessment

    The Functional Assessment of Cancer Therapy - Anemia (FACT-An) is the Health-Related Quality of Life (HRQoL) tool used in this study. The lowest possible score is 0, which reflects the worst possible quality of life, indicating severe impairments in overall well-being and significant anemia-related symptoms. The highest possible score is 188, representing the best possible quality of life, with minimal or no symptoms and optimal functioning across all domains, including anemia-related concerns.

    Measured: 24 Weeks

  25. Mean Change Health-related quality of life (HRQoL) Assessment

    The Functional Assessment of Cancer Therapy - Anemia (FACT-An) is the Health-Related Quality of Life (HRQoL) tool used in this study. The lowest possible score is 0, which reflects the worst possible quality of life, indicating severe impairments in overall well-being and significant anemia-related symptoms. The highest possible score is 188, representing the best possible quality of life, with minimal or no symptoms and optimal functioning across all domains, including anemia-related concerns.

    Measured: 48 Weeks

Timeline

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

  1. First posted23 Jan 2025
  2. Study start25 Mar 2025
  3. Primary completionFeb 2027 (estimated)
  4. Study completionFeb 2028 (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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06788691
Sponsor's protocol ID
24-06027622
Status last verified
Mar 2026
First submitted
17 Jan 2025
Last updated
24 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Luspatercept in people with CCUS Clonal Cytopenia of Undetermined Significance and Anemia, run by Weill Medical College of Cornell University 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 22 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 24 Mar 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?

Contact Pinkal Desai, MD (646-962-2700, pid9006@med.cornell.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients achieving cytopenia responses as defined by HI-E, HI-P, HI-N, mHI-E responses (time frame: 24 Weeks). 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 NCT06788691.

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 24 Mar 2026.

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