Myelodysplastic SyndromesNCT06851065
A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States
An observational study of Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 1 site in United States.
Trial summary
The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Luspatercept and Erythropoiesis-stimulating agents
- How many people
- 418 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Aug 2024to 10 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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
Study population: The study population will include adults in the United States with a clinician-confirmed diagnosis of primary or secondary lower-risk myelodysplastic syndromes (LR-MDS) who initiated first-line luspatercept treatment on or after LR-MDS diagnosis date between 28 August 2023 to 31 July 2024
Who may be able to join
- Had a documented diagnosis of primary or secondary myelodysplastic syndromes (MDS)
- MDS diagnosis confirmed through bone marrow testing on (or 30 days prior to) MDS diagnosis date or within 1 year of MDS diagnosis date
- Had a documented determination of Lower Risk (LR)-MDS as measured by International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) at or before index treatment (i.e., first-line luspatercept or first-line erythropoiesis-stimulating agents (ESA)) initiation
- IPSS risk level: low, intermediate-1 (level-1 risk)
- IPSS-R risk level: very low, low, intermediate
- Received luspatercept as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 1)
Show all 13Show fewer
- Receipt of combination therapy with ESAs and/or granulocyte colony-stimulating factors (G-CSFs) will be allowed
- OR
- Received ESA as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 2)
- Was aged 18 years or older at the time of initial diagnosis of MDS
- Known vital status (i.e., living, or deceased) at the time of record abstraction.
- Records for patients who are dead or alive will be eligible
- Complete medical record covering relevant past medical history, diagnosis of LR-MDS, treatment, laboratory assessments, red-blood cell (RBC) transfusions, and regular monitoring for LR-MDS, including any transfer record from other physicians/facilities (if applicable) is available to the abstracting physician for data abstraction
Who cannot join
- Had a history of acute myeloid leukemia (AML) prior to MDS diagnosis
- Received previous treatment with hypomethylating agents, disease-modifying agents (including lenalidomide), other immunosuppressants/immunomodulatory agents, or other MDS-directed chemotherapy
- Received stem cell transplant prior to index treatment initiation
- Participated in a clinical trial for the treatment of MDS before or while on index treatment (i.e., clinical trial participation after first-line luspatercept or ESA treatment discontinuation will be allowed)
- Had evidence of other malignant neoplasms in the 12 months prior to diagnosis of MDS, except basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histologic finding of prostate cancer (stage T1a or T1b)
- Patients for whom this information is not available (i.e., "unknown") will be included in the study
- For Cohort 1 (i.e., first-line luspatercept treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
- For Cohort 2 (i.e., first-line ESA treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, luspatercept, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
Trial locations and contacts
1 site in 1 country.
United States1 site
RTI Health Solutions
Raleigh, North Carolina, 27709-2194
About this study
Official title: Luspatercept Treatment Patterns and Outcomes Among ESA-Naïve Patients With Lower-Risk MDS - A Retrospective Medical Record Review in the United States
The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Luspatercept, Erythropoiesis-stimulating agents
- Keywords
- Lower-risk myelodysplastic syndromes (LR-MDS)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuspaterceptDrug
As per product lable
Erythropoiesis-stimulating agentsDrug
As per product label
| Study group | Role | What they receive |
|---|---|---|
| Participants receiving first-line luspatercept treatment | Not described Receives: Drug: Luspatercept | |
| Participants receiving first-line erythropoiesis-stimulating agents | Not described Receives: Drug: Erythropoiesis-stimulating agents |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participant baseline demographics
Measured: Baseline
Participant baseline clinical characteristics
Measured: Baseline
Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation
Measured: Baseline
Rationale for therapy selection
Measured: Baseline
Duration of index treatment
Measured: Up to 15 months
Treatment dose at treatment initiation and discontinuation
Measured: Up to 6 months
Treatment dose/dosing schedule changes, and treatment interruptions
Measured: Up to 12 months
Other supportive care therapies prescribed while on index treatment
Measured: Up to 15 months
Treatments prescribed post index treatment
Measured: Up to 15 months
Treatments for anemia management received after discontinuing the index treatment
Measured: Up to 15 months
Receipt of stem cell transplant at any time post index treatment
Measured: Up to 15 months
Participant red-blood cell (RBC) transfusion burden post index treatment
Measured: At 3-months, and up to 6 months
Hematologic improvement-erythroid (HI-E) response post index treatment
Measured: At 3-months, and up to 6 months
Progression to acute myeloid leukemia post index treatment
Measured: Up to 15 months
Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria
Measured: Up to 15 months
Participant adverse events during and post index treatment
Measured: Up to 15 months
Overall survival (OS)
Measured: At 3-, 6-, 12-, and up to 15-months
Healthcare resource utilization (HCRU) during index treatment
Measured: Up to 15 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2025
- Study start22 Aug 2024
- Primary completion10 Mar 2025
- Study completion10 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 418 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06851065
- Sponsor's protocol ID
- CA056-1121
- Status last verified
- Feb 2026
- First submitted
- 24 Feb 2025
- Last updated
- 17 Feb 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Luspatercept Treatment Patterns and Outcomes Among ESA-Naïve Patients With Lower-Risk MDS - A Retrospective Medical Record Review in the United States
- ClinicalTrials.gov ID
- NCT06851065
- Acronym / sponsor ID
- CA056-1121
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Lower-risk myelodysplastic syndromes (LR-MDS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 418 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 22 Aug 2024 (actual)
- Primary completion
- 10 Mar 2025 (actual)
- Study completion
- 10 Mar 2025 (actual)
- First posted
- 28 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Participants receiving first-line luspatercept treatment | – | Drug: Luspatercept |
| Participants receiving first-line erythropoiesis-stimulating agents | – | Drug: Erythropoiesis-stimulating agents |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will include adults in the United States with a clinician-confirmed diagnosis of primary or secondary lower-risk myelodysplastic syndromes (LR-MDS) who initiated first-line luspatercept treatment on or after LR-MDS diagnosis date between 28 August 2023 to 31 July 2024
Key inclusion criteria (13 listed)
- Had a documented diagnosis of primary or secondary myelodysplastic syndromes (MDS)
- MDS diagnosis confirmed through bone marrow testing on (or 30 days prior to) MDS diagnosis date or within 1 year of MDS diagnosis date
- Had a documented determination of Lower Risk (LR)-MDS as measured by International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) at or before index treatment (i.e., first-line luspatercept or first-line erythropoiesis-stimulating agents (ESA)) initiation
- Received luspatercept as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 1)
- OR
Key exclusion criteria (8 listed)
- Had a history of acute myeloid leukemia (AML) prior to MDS diagnosis
- Received previous treatment with hypomethylating agents, disease-modifying agents (including lenalidomide), other immunosuppressants/immunomodulatory agents, or other MDS-directed chemotherapy
- Received stem cell transplant prior to index treatment initiation
- Participated in a clinical trial for the treatment of MDS before or while on index treatment (i.e., clinical trial participation after first-line luspatercept or ESA treatment discontinuation will be allowed)
- Had evidence of other malignant neoplasms in the 12 months prior to diagnosis of MDS, except basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histologic finding of prostate cancer (stage T1a or T1b)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LuspaterceptAs per product lable | Drug | Participants receiving first-line luspatercept treatment |
| Erythropoiesis-stimulating agentsAs per product label | Drug | Participants receiving first-line erythropoiesis-stimulating agents |
Primary outcome
Participant baseline demographics
Time frame: Baseline
Participant baseline clinical characteristics
Time frame: Baseline
Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation
Time frame: Baseline
Rationale for therapy selection
Time frame: Baseline
Duration of index treatment
Time frame: Up to 15 months
Treatment dose at treatment initiation and discontinuation
Time frame: Up to 6 months
Treatment dose/dosing schedule changes, and treatment interruptions
Time frame: Up to 12 months
Other supportive care therapies prescribed while on index treatment
Time frame: Up to 15 months
Treatments prescribed post index treatment
Time frame: Up to 15 months
Treatments for anemia management received after discontinuing the index treatment
Time frame: Up to 15 months
Receipt of stem cell transplant at any time post index treatment
Time frame: Up to 15 months
Participant red-blood cell (RBC) transfusion burden post index treatment
Time frame: At 3-months, and up to 6 months
Hematologic improvement-erythroid (HI-E) response post index treatment
Time frame: At 3-months, and up to 6 months
Progression to acute myeloid leukemia post index treatment
Time frame: Up to 15 months
Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria
Time frame: Up to 15 months
Participant adverse events during and post index treatment
Time frame: Up to 15 months
Overall survival (OS)
Time frame: At 3-, 6-, 12-, and up to 15-months
Healthcare resource utilization (HCRU) during index treatment
Time frame: Up to 15 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
- BMS Clinical Trial InformationLink provided by the sponsor.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingPhase 2
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
Myelodysplastic Syndrome
Enrolling by invitationPhase 1
A Rollover Study for Continued Study Treatment and Ongoing Safety Monitoring
Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Chronic, B-Cell
RecruitingPhase 1
First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)
R/R AML, R/R HR-MDS
RecruitingObservational study
Natural History of Trisomy 8-Associated Autoinflammatory Disease (TRIAD) and Related Disorders
Trisomy 8 Mosaicism, Trisomy 8 Associated Autoinflammatory Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bristol Myers Squibb study director
Bristol-Myers Squibb
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes, run by Bristol-Myers Squibb 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 13 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: finished, as of the sponsor's last update on 17 Feb 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Participant baseline demographics (time frame: Baseline). The study lists 18 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Feb 2026; AzTrials last refreshed its copy on 5 Nov 2026.