RecruitingPhase 1 / Phase 2NCT06303193
Blood
Aplastic Anemia
Also known as bone marrow failure, acquired aplastic anemia, AA
Aplastic anemia is a life-threatening bone marrow failure syndrome characterized by pancytopenia resulting from destruction or suppression of hematopoietic stem cells, most commonly by autoreactive T lymphocytes. The acquired form is typica
ORPHA:87806 ↗Prevalence 2-3 per million per year in Western countries; 2-3 times higher in East AsiaOnset Any age; bimodal peaks in young adults (15-25) and older adults (>60)Acquired (immune-mediated) or inherited
69
studies recruiting now
as of 7 Sept 2026
511
studies registered in total
as of 7 Sept 2026
1
countries with a recruiting site
as of 7 Sept 2026
20 Dec 2024
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2NCT03520647
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
RecruitingNCT05687149
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
RecruitingNCT00027274
Cancer in Inherited Bone Marrow Failure Syndromes
RecruitingNCT06744283
Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 69 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
Keep watching
Get an email when a new Aplastic Anemia study opens.
One email a day at most. Unsubscribe with one click.
Used only for these alerts. Privacy.
Support
Patient organisations
Registry: AA/MDS International Foundation Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Aplastic Anemia
Aplastic anemia is a life-threatening bone marrow failure syndrome characterized by pancytopenia resulting from destruction or suppression of hematopoietic stem cells, most commonly by autoreactive T lymphocytes. The acquired form is typically immune-mediated and may be triggered by viral infections, toxic exposures, or medications, though most cases are idiopathic. Treatment ranges from immunosuppressive therapy with antithymocyte globulin and cyclosporine to allogeneic hematopoietic stem cell transplantation in eligible patients.
Common clinical features
Severe fatigue and pallor from anemiaIncreased susceptibility to infections from neutropeniaEasy bruising and bleeding from thrombocytopeniaPetechiae and mucosal bleedingShortness of breath with minimal exertionRecurrent infections including bacterial and fungalBone marrow biopsy showing hypocellular marrow with fat replacementAbsence of organomegaly or lymphadenopathy (differentiating from leukemia)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
10 approved treatments and 15 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Oxymetholone (Adroyd)Approved: Antilymphocyte Immunoglobulin (Horse)Approved: Dexamethasone Sodium Phosphate (Ak-dex)Approved: Prednisolone (Cortalone)Approved: Filgrastim (Accofil)Approved: Dexamethasone (Aeroseb-dex)Approved: Eltrombopag Choline (Alvaiz)Approved: Cortisone Acetate (Cortate)Approved: Prednisone (Cortan)Approved: Muplestim (Hemokine)
Phase 3Cyclophosphamide (Cyclophosphamide)
Phase 2/3Eltrombopag (Revolade)
Phase 2/3Romiplostim (Nplate)
Phase 2/3Avatrombopag
Phase 2Decitabine (Dacogen)
Phase 2Rituximab (Blitzima)
Phase 2Levamisole
Phase 2Acetylcysteine (A-cys)
+ 7 more in development
Before you apply
Things trial teams commonly ask about for Aplastic Anemia. Not eligibility rules; those are set by each study.
- Severity classification (moderate, severe, or very severe AA based on neutrophil, platelet, and reticulocyte counts) determines trial eligibility; obtain a recent complete blood count and bone marrow biopsy report.
- Prior immunosuppressive therapy history (IST) including ATG cycles, cyclosporine use, and response status is critical as most trials stratify by treatment-naive versus relapsed/refractory status.
- Rule out inherited bone marrow failure syndromes (Fanconi anemia, dyskeratosis congenita) before trial enrollment, as many trials exclude these; telomere length testing and chromosome fragility assays may be required.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).