Adults 2 to 100, any sex, with Severe Aplastic Anemia (SAA). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Drug Response as Defined by Clinically-signficant Hematologic ImprovementsPrimary· 24 weeks
Defined as unilineage or multilineage recovery by 1 or more of the following: 1) platelet response (increase to 20 × 103/μL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks in those who were transfusion dependent on entry into the protocol); (2) erythroid response (when pretreatment hemoglobin was \<9 g/dL, defined as an increase in hemoglobin by 1.5 g/dL or, in transfused patients, a reduction in the units of packed red blood cell transfusions by an absolute number of at least 4 transfusions for 8 consecutive weeks, compared with the pretreatment
Group
Value
95% CI
Eltrombopag
20
Adverse events — posted to ClinicalTrials.gov
Time frame: 6 months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Eltrombopag
Serious: 13/40 (33%)
Deaths: 1/40
Serious adverse events (27 terms)
Reaction
System
Eltrombopag
Febrile neutropenia
Blood and lymphatic system disorders
—
Sepsis
Infections and infestations
—
Anaemia
Blood and lymphatic system disorders
—
Otitis media
Ear and labyrinth disorders
—
Colitis
Gastrointestinal disorders
—
Diarrhoea
Gastrointestinal disorders
—
Nausea
Gastrointestinal disorders
—
Pancreatitis
Gastrointestinal disorders
—
Vomiting
Gastrointestinal disorders
—
Death
General disorders
—
Infusion related reaction
General disorders
—
Pyrexia
General disorders
—
Bile duct stenosis
Hepatobiliary disorders
—
Hypersensitivity
Immune system disorders
—
Urticaria
Immune system disorders
—
Cellulitis
Infections and infestations
—
Clostridium difficile Infection
Infections and infestations
—
Influenza
Infections and infestations
—
Staphylococcal infection
Infections and infestations
—
Craniocerebral Injury
Injury, poisoning and procedural complications
—
Haemorrhage intracranial
Nervous system disorders
—
Headache
Nervous system disorders
—
Nephrolithiasis
Renal and urinary disorders
—
Ovarian haemorrhage
Reproductive system and breast disorders
—
Epistaxis
Respiratory, thoracic and mediastinal disorders
—
Other adverse events (180 terms — click to expand)
Background:
\- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia.
Objectives:
\- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts.
Eligibility:
\- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.
Design:
* Participants will take eltrombopag by mouth once a day for 6 months.
* Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow.
* Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07189910 — Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP
· Phase 4
· not yet recruiting
NCT07113743 — Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
· Phase 1, PHASE2
· enrolling by invitation
NCT07093606 — Eltrombopag Induced Liver Dysfunction During Treatment of Immunethrombocytopenic Purpura
· NA
· recruiting
NCT06345495 — High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
· Phase 2
· recruiting
NCT05653219 — A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia
· Phase 3
· active not recruiting
Other recruiting trials for Severe Aplastic Anemia (SAA)
Currently open trials in the same condition.
NCT03520647 — Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells a
· Phase 2
· recruiting
Other National Heart, Lung, and Blood Institute (NHLBI) trials
Trials by the same sponsor.
NCT07566494 — Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
· Phase 1
· not yet recruiting
NCT07137455 — EDEN Intracardiac Electrogram Recording and Classifying System
· NA
· enrolling by invitation
NCT07516379 — GRAfT 2.0. A Multimodal Prospective Approach to Define the Mechanisms and Clinical Features of Acute and Chronic Rejecti
· not yet recruiting
NCT06948097 — Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fost
· Phase 1
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
Last refreshed: 25 April 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01891994.