Myelodysplastic SyndromesNCT07681440
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia
An observational study of Myelodysplastic Syndromes and Beta-thalassemia, run by Bristol-Myers Squibb at 1 site in Germany.
Trial summary
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Beta-thalassemia
- What is being tested?
- Luspatercept
- How many people
- 30 participants
- Where
- 1 siteGermany
- Study dates
- 16 Nov 2023to 1 May 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 adult patients diagnosed with myelodysplastic syndromes or beta-thalassemia and treated with luspatercept between January 1, 2020, to June 30, 2022, identified from a German Statutory Health Insurance database
Who may be able to join
- Participants treated with luspatercept. The date of first treatment with luspatercept will be assigned as the index date.
- At least one Lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia diagnosis in inpatient or at least two MDS or beta-thalassemia diagnosis in the outpatient setting at least 30 days apart prior to or at index date.
- 18+ years old at index date.
- Participants with a minimum of 12-months observable pre-treatment baseline/look-back prior to index date.
- Participants with a minimum of 6-months of follow-up time from the index date unless the patient is lost due to death
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting
- Conditions
- Myelodysplastic Syndromes, Beta-thalassemia
- Being tested
- Luspatercept
- Keywords
- Lower-risk myelodysplastic syndromes (LR-MDS), Beta-thalassemia
- Medical subject headings
- Myelodysplastic Syndromes, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuspaterceptDrug
According to the product label
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Participants diagnosed myelodysplastic syndromes receiving luspatercept treatment Receives: Drug: Luspatercept | |
| Cohort 2 | Participants diagnosed beta-thalassemia receiving luspatercept treatment Receives: Drug: Luspatercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participant age
Measured: Baseline
Participant gender
Measured: Baseline
Year of first luspatercept prescription
Measured: Baseline
Number of participants by Ring Sideroblast (RS) status (positive/negative/unknown) as measured by bone marrow aspiration
As documented in participant medical charts
Measured: Baseline
Participant comorbidities
Measured: Baseline
Length of follow-up from start of luspatercept treatment initiation until the earliest of the end of continuous enrolment, death, or end of data availability
Measured: Baseline
Secondary outcomes (4)
Number of participants that achieve red blood cell (RBC) transfusion independence
Number of participants in Cohort 1 achieving RBC transfusion independence, defined as no RBC transfusions during any consecutive 56-day (8-week) or 84-day (12-week) period within the first 168 days (24 weeks) after the index date, as assessed from transfusion records and medical chart review.
Measured: Up to 12 months
Total number of RBC units transfused per participant, as assessed from transfusion records and medical chart review, from baseline up to 6 months
Measured: Baseline and up to 6 months
Time to acute myeloid leukemia transformation
Measured: Up to 6 months
Time to treatment discontinuation
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2026
- Study start16 Nov 2023
- Primary completion1 May 2025
- Study completion1 May 2025
Why it stopped: Insufficient enrollment
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
- 30 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
- NCT07681440
- Sponsor's protocol ID
- CA056-1082
- Status last verified
- Jun 2026
- First submitted
- 10 Oct 2025
- Last updated
- 2 Jul 2026
- Results posted
- No
- Data monitoring committee
- 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 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients
- ClinicalTrials.gov ID
- NCT07681440
- Acronym / sponsor ID
- CA056-1082
- Conditions
- Myelodysplastic Syndromes, Beta-thalassemia
- Keywords
- Lower-risk myelodysplastic syndromes (LR-MDS), Beta-thalassemia
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Insufficient enrollment
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 16 Nov 2023 (actual)
- Primary completion
- 1 May 2025 (actual)
- Study completion
- 1 May 2025 (actual)
- First posted
- 2 Jul 2026 (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 the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: LuspaterceptParticipants diagnosed myelodysplastic syndromes receiving luspatercept treatment |
| Cohort 2 | – | Drug: LuspaterceptParticipants diagnosed beta-thalassemia receiving luspatercept treatment |
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 adult patients diagnosed with myelodysplastic syndromes or beta-thalassemia and treated with luspatercept between January 1, 2020, to June 30, 2022, identified from a German Statutory Health Insurance database
Key inclusion criteria (5 listed)
- Participants treated with luspatercept. The date of first treatment with luspatercept will be assigned as the index date.
- At least one Lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia diagnosis in inpatient or at least two MDS or beta-thalassemia diagnosis in the outpatient setting at least 30 days apart prior to or at index date.
- 18+ years old at index date.
- Participants with a minimum of 12-months observable pre-treatment baseline/look-back prior to index date.
- Participants with a minimum of 6-months of follow-up time from the index date unless the patient is lost due to death
Key exclusion criteria (1 listed)
- None
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 |
|---|---|---|
| LuspaterceptAccording to the product label | Drug | Cohort 1, Cohort 2 |
Primary outcome
Participant age
Time frame: Baseline
Participant gender
Time frame: Baseline
Year of first luspatercept prescription
Time frame: Baseline
Number of participants by Ring Sideroblast (RS) status (positive/negative/unknown) as measured by bone marrow aspiration
Time frame: Baseline
As documented in participant medical charts
Participant comorbidities
Time frame: Baseline
Length of follow-up from start of luspatercept treatment initiation until the earliest of the end of continuous enrolment, death, or end of data availability
Time frame: Baseline
4 secondary outcomes
Number of participants that achieve red blood cell (RBC) transfusion independence
Time frame: Up to 12 months
Total number of RBC units transfused per participant, as assessed from transfusion records and medical chart review, from baseline up to 6 months
Time frame: Baseline and up to 6 months
Time to acute myeloid leukemia transformation
Time frame: Up to 6 months
Time to treatment discontinuation
Time frame: Up to 6 months
Results and safety
The study was stopped early (reason given: Insufficient enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2026.
- BMS Clinical Trial InformationLink provided by the sponsor.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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 and Beta-thalassemia, run by Bristol-Myers Squibb at 1 site in Germany.
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 5 inclusion and 1 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: stopped early, as of the sponsor's last update on 2 Jul 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 Germany. 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 was stopped early (reason given: Insufficient enrollment). Results are not available in the source record.
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 age (time frame: Baseline). The study lists 6 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 2 Jul 2026.