How does a blood disorder drug perform in everyday practice?
NCT ID NCT07681440
First seen Jul 02, 2026 · Last updated Jul 02, 2026
Summary
This study looks at how the drug luspatercept is used in real-world clinics for patients with lower-risk myelodysplastic syndromes (MDS) or beta-thalassemia. Researchers will track patient characteristics, treatments, and outcomes like reduced need for blood transfusions. The goal is to understand the drug's effectiveness and costs outside of strict clinical trial settings.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- luspatercept
- What this could lead to
- If successful, this could help doctors understand how luspatercept works outside of clinical trials, potentially improving treatment guidelines for these blood disorders.
- What could go wrong
- This is an observational study, not a controlled trial, so it cannot prove cause and effect. Results may be limited by small size and real-world data variability.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
30 people
The number who actually took part.
- Started
-
Nov 2023
- Finished
-
May 2025
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
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
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Exclusion Criteria: • None
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
ZEG Berlin, Center for Epidemiology and Health Research
Berlin, Germany
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