New hope for tough anemia: drug boosts blood cells in failed cases
NCT ID NCT03988608
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called eltrombopag in 20 Chinese adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants had not responded to previous treatments. The drug was given daily for up to a year to see if it could improve blood cell counts and reduce the need for transfusions. The goal was to control the disease, not cure it.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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20 people
The number who actually took part.
- Started
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Dec 2019
- Finished
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May 2023
- Lead sponsor
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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.
- Ages
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18 to 100 years
- Sex
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Anyone
- Healthy volunteers
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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: * Chinese patients aged greater than or equal to 18 years old. * Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of \> 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab. * Platelet count ≤ 30 × 10\^9/L at screening. * Patient must not currently have the option of stem cell transplantation. * Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2. * Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or \<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site. Exclusion Criteria: * Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months. * Congenital aplastic anemia * AST or ALT ≥3 times the upper limit of normal. * Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome). * Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%. * Presence of chromosomal aberration (-7/7q- detected by fluorescence in situ hybridization (FISH), or other aberrations detected by G-band staining). * Evidence of a clonal hematologic bone marrow disorder on cytogenetics. * Past medical history of thromboembolism within 6 months or current use of anticoagulants. * Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years. * Patient with clinically significant (of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy) bacterial, fungal, mycobacterial, parasitic or viral infection (Patient with acute bacterial infections requiring antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed). * Patient with known hepatocellular disease * Presences of hepatitis B surface antigen (HBsAg), positive hepatitis C antibody test result at screening. * Cardiac disorder (NYHA) functional classification Grade II/III/IV * Past medical history of immediate or delayed hypersensitivity to compounds chemically similar to eltrombopag or their excipients. * Treatment with another investigational product within 30 days. * Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist. * Positive result for HIV (Human Immunodeficiency Virus) antibody test. * Pregnant or nursing (lactating) woman. * Woman of child-bearing potential.
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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
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Novartis Investigative Site
Nanjing, Jiangsu, 210000, China
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Novartis Investigative Site
Nanchang, Jiangxi, 330006, China
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Novartis Investigative Site
Chengdu, Sichuan, 610041, China
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Novartis Investigative Site
Tianjin, 300020, China
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Novartis Investigative Site
Tianjin, 300052, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as a cure for severe aplastic anemia
- 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?