Promising combo for aplastic anemia trial pulled before start
NCT ID NCT06424639
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial aimed to compare the safety and effectiveness of adding luspatercept to cyclosporine versus cyclosporine alone in adults newly diagnosed with non-severe aplastic anemia (NSAA). The goal was to see if the combination could improve blood cell counts better than the standard drug alone. However, the study was withdrawn before enrolling any participants, so no results are available.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- luspatercept and cyclosporine
- What this could lead to
- If it had worked, this could have pointed toward a more effective treatment for non-severe aplastic anemia, potentially improving blood cell counts without transfusions.
- What could go wrong
- The trial was withdrawn before any patients were enrolled, so no results are available. Even if conducted, it was a small early-phase study, and the combination may not have proven safer or more effective than cyclosporine alone.
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.
- Phase
-
Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Expected to start
-
May 2024
An estimate. Start dates often move.
- Expected to finish
-
Dec 2025
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 90 years
- 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: 1. Age ≥ 18 years 2. Hemoglobin level of 6-10 g/dL 3. Definition of NSAA: Patients with AA diagnosis but not SAA or VSAA diagnosis (at least two of the following conditions can be diagnosed as AA: (i) Hemoglobin \< 100 g/L; (ii) Platelet count \< 50×10\^9/L; (iii) Neutrophil count \< 1.5×10\^9/L. SAA diagnosis criteria include less than 25% (or 25-50%, but residual hematopoietic cells \< 30%) of bone marrow cells, plus at least two of the following conditions: (i) Neutrophil count \< 0.5×10\^9/L; (ii) Platelet count \< 20×10\^9/L; (iii) Retroperitoneal lymph node count \< 20×10\^9/L. VSAA meets the criteria for SAA, but with neutrophil count \< 0.2×10\^9/L. (British guidelines, 2015)) 4. No active infection 5. No other concurrent neoplasms (except in situ carcinoma) 6. Baseline liver and renal function within 1.5 times of normal value 7. No pregnancy or breastfeeding 8. Agree to sign informed consent form 9. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. Exclusion Criteria: 1. Congenital aplastic anemia 2. Presence of chromosomal aberrations 3. Cytogenetic evidence of clonal hematological myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML) 4. PNH clone ≥50% 5. Previous use of alemtuzumab, any ATG, or any dose of cyclosporine for immunosuppressive treatment 6. Previous hematopoietic stem cell transplant (HSCT) 7. Uncontrolled infection or bleeding under standard treatment 8. Allergy to rituximab, cyclosporine, or excipients 9. History of allergy to polyethylene glycol (PEG) 80 10. Active infection or cirrhosis of the liver or portal hypertension due to HIV, HCV, or HBV 11. Screening QTcF (Fridericia QT corrected formula) less than 450 milliseconds or less than 480 milliseconds of bundle branch block determined by three ECG averages, and assessed on-site; unstable angina; uncontrolled hypertension (\>180/100 mmHg); pulmonary hypertension 12. Any malignant tumor within 5 years, except local basal cell carcinoma; previous thromboembolic event, history of myocardial infarction or stroke (including antiphospholipid syndrome); currently using anticoagulants 13. Pregnant or lactating women 14. Participated in another clinical trial within 3 months
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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
-
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a pill revive bone marrow in aplastic anemia?
- Can a drug revive blood cell production in children with aplastic anemia?
- Could a platelet-boosting drug shorten recovery after stem cell transplants?
- A common antioxidant may help platelets recover faster after stem cell transplant
- Could a cancer antibody reboot blood production in aplastic anemia?