Blood

Idiopathic aplastic anemia

ORPHA:88 ↗Gene PRF1 Gene SBDS Gene IFNG Gene TERT Gene TERC

Search all Idiopathic aplastic anemia trialsAlert me when a trial opens

studies recruiting now

as of 7 Sept 2026

369

studies registered in total

as of 7 Sept 2026

countries with a recruiting site

as of 7 Sept 2026

12 Mar 2024

most recent study posted

among recruiting studies

Recruiting trials

RecruitingPhase 1 / Phase 2NCT06303193

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Sponsor National Cancer Institute (NCI)Where United States (1 site)Studying pacritinibUpdated 4 Sept 2026

See details

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

Sponsor National Heart, Lung, and Blood Institute (NHLBI)Where United States (1 site)Studying Cyclophosphamide, Peripheral Blood Stem CellsUpdated 1 Sept 2026

See details

RecruitingNCT00027274

Cancer in Inherited Bone Marrow Failure Syndromes

Sponsor National Cancer Institute (NCI)Where United States (2 sites)Updated 31 Aug 2026

See details

RecruitingNCT05012111

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Sponsor National Heart, Lung, and Blood Institute (NHLBI)Where United States (1 site)Updated 27 Aug 2026

See details

RecruitingPhase 1 / Phase 2NCT03173937

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Sponsor National Heart, Lung, and Blood Institute (NHLBI)Where United States (1 site)Studying Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent SystemUpdated 26 Aug 2026

See details

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 51 recruiting studies

Where 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.

United States

Keep watching

Get an email when a new Idiopathic aplastic anemia study opens.

One email a day at most. Unsubscribe with one click.

Used only for these alerts. Privacy.

Support

Patient organisations

We do not yet list a dedicated organisation for this condition. The directories below are the best route.

NORD ↗GARD ↗Orphanet ORPHA:88 ↗

About Idiopathic aplastic anemia

RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the genes involved (PRF1, SBDS, IFNG, TERT, TERC).

Orphanet expert page ↗GARD (NIH) ↗

Treatments being studied

2 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Phase 3Eltrombopag (Revolade)

Phase 2Cyclophosphamide (Cyclophosphamide)

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).