Anemia, Aplastic clinical trials
The registry lists 371 studies for Anemia, Aplastic across more than 10 countries. 98 are recruiting or about to start. 235 of them are mainly about Anemia, Aplastic (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10. Counting its 3 narrower conditions too, such as Fanconi Anemia: 510 studies, 120 open.
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· Sites: 4 added, including Seoul and Busan
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Status
Who runs them
National Heart, Lung, and Blood Institute (NHLBI) (41) · Institute of Hematology & Blood Diseases Hospital, China (29) · Peking Union Medical College Hospital (22) · Novartis Pharmaceuticals (14) · Peking University People's Hospital (10) · M.D. Anderson Cancer Center (8) · Jiangsu HengRui Medicine Co., Ltd. (7) · Roswell Park Cancer Institute (7)
What is being tested here
Drugs under test in Anemia, Aplastic studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Drug classes tested here Alkylating agents 9 open · Calcineurin inhibitors 4 open · Tyrosine kinase inhibitors (TKIs) 4 open · JAK inhibitors 4 open · Corticosteroids 3 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The 10 nearest
- Nov 2026Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy · Washington University School of Medicine
- Dec 2026Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita · Boston Children's Hospital
- Dec 2026Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness · Institute of Hematology & Blood Diseases Hospital, China
- Dec 2026Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA · Chinese PLA General Hospital
- Dec 2026Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia · Institute of Hematology & Blood Diseases Hospital, China
- Dec 2026AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion · Mitchell Cairo
- Jan 2027Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders · Masonic Cancer Center, University of Minnesota
- Jan 2027Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression · Assistance Publique - Hôpitaux de Paris
- Jan 2027ATGAM General Investigation · Pfizer
- Feb 2027French National Registry of Bone Marrow Failures · Assistance Publique - Hôpitaux de Paris
Who these studies take
Of the 58 Anemia, Aplastic studies recruiting now, 20 (34%) set no upper age limit and 30 (52%) stop at 75 or younger. 37 (64%) take children under 18. 1 (2%) are for women only and 0 (0%) for men only; 5 (9%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 32 (55%) | 32 (55%) |
| 18 | 52 (90%) | 52 (90%) |
| 30 | 50 (86%) | 49 (84%) |
| 40 | 49 (84%) | 48 (83%) |
| 50 | 47 (81%) | 46 (79%) |
| 65 | 43 (74%) | 43 (74%) |
| 75 | 38 (66%) | 38 (66%) |
| 85 | 26 (45%) | 26 (45%) |
Across all 371 studies, recruiting or not: 49% stop at 75 or younger and 33% at 65; 63% take children; 1% are for women only and 1% for men only; 6% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Anemia, Aplastic studies take
Of 60 phase 2 interventional studies under Anemia, Aplastic, half reached primary completion within 76 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Phase 2 | 76 mo39–104 · 60 studies | — | 93 mo55–111 · 52 studies |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 13, 2026.
Started recruiting2
Results posted1
- Low-Dose Danazol for the Treatment of Telomere Related DiseasesOct 27, 2026NCT03312400
Stopped5
- CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic AnemiaSuspendedNov 4, 2026NCT06622694
- Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic AnemiaTerminatedNov 4, 2026NCT06695741
- Low-Dose Danazol for the Treatment of Telomere Related DiseasesTerminatedOct 27, 2026NCT03312400
- A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)TerminatedOct 19, 2026NCT05600426
- UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic AnemiaWithdrawnOct 16, 2026NCT07001254
Newly registered1
- Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBTOct 21, 2026NCT07885982
Studies
Recruiting first, then most recently updated. Showing 50 of 371. Browse all 371 → or search them.
- 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 NeoplasmsRecruitingNCT06303193Phase 1/21 country
- Cancer in Inherited Bone Marrow Failure SyndromesRecruitingNCT000272741 country
- Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsRecruitingNCT01174108Phase 21 country
- HPC Offered for PRESERVE ExpansionRecruitingNCT071454111 country
- The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic AnemiaRecruitingNCT07287228Phase 1/21 country
- A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure SyndromesRecruitingNCT07712562Phase 1/21 country
- Natural History of Acquired and Inherited Bone Marrow Failure SyndromesRecruitingNCT050121111 country
- Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisRecruitingNCT03520647Phase 21 country
- Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic CancerRecruitingNCT011376431 country
- AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell DepletionRecruitingNCT04099966Phase 21 country
- Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBTNot yet recruitingNCT07885982Phase 21 country
- CK0801 for Treatment of Aplastic AnemiaNot yet recruitingNCT07499102Phase 2
- Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research StudyEnrolling by invitationNCT071028491 country
- Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic AnemiaRecruitingNCT02828592Phase 21 country
- Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Bone Marrow or Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific AntibodiesRecruitingNCT06398457Early Phase 11 country
- 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 OutcomeRecruitingNCT03173937Phase 1/21 country
- Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic AnemiaRecruitingNCT07714512Phase 1/21 country
- Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic AnemiaNot yet recruitingNCT07819812
- Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRSRecruitingNCT075786751 country
- Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia PatientsNot yet recruitingNCT07737262Phase 3
- Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic AnemiaRecruitingNCT07629947Not applicable1 country
- Haploidentical HCT for Severe Aplastic AnemiaRecruitingNCT04558736Phase 21 country
- Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNINot yet recruitingNCT07796620Phase 1/2
- The HOPE Biobank Resource (BMT CTN 2402 HOPE)RecruitingNCT072271551 country
- A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard TreatmentNot yet recruitingNCT07786922Phase 2/3
- Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell TransplantationNot yet recruitingNCT07698080Phase 2
- A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)RecruitingNCT013515451 country
- A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAARecruitingNCT075867351 country
- TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in ChildrenRecruitingNCT04356469Phase 21 country
- Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAAEnrolling by invitationNCT07299123Phase 1/21 country
- Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic SyndromeRecruitingNCT01624805Phase 21 country
- Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular TherapyRecruitingNCT06689800Not applicable1 country
- MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.RecruitingNCT06412497Phase 21 country
- Romiplostim N01 in Chemoradiotherapy-induced Aplastic AnemiaNot yet recruitingNCT07572084Phase 2
- CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAANot yet recruitingNCT07558434Phase 2
- Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 YearsRecruitingNCT06646497Phase 21 country
- Clinical Study on the Preservation of Ovarian Function in Patients With Aplastic Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation Using GoserelinNot yet recruitingNCT07552506Not applicable1 country
- Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAANot yet recruitingNCT07548671Phase 3
- Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AARecruitingNCT07400341Phase 21 country
- Managed Access Programs for ETB115, EltrombopagAvailableNCT04837703
- A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic AnemiaRecruitingNCT07499284Not applicable1 country
- Adverse Effects of ATG/ALG Therapy in Aplastic AnemiaRecruitingNCT074611161 country
- Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)RecruitingNCT04304820Phase 21 country
- Revolade Tablets Specified Drug-use SurveyRecruitingNCT062872681 country
- Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic AnemiaRecruitingNCT06517641Phase 21 country
- National Longitudinal Cohort of Hematological DiseasesRecruitingNCT046451991 country
- Alpha/Beta TCD HCT in Patients With Inherited BMF DisordersRecruitingNCT03579875Phase 21 country
- A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic AnemiaNot yet recruitingNCT07345000Phase 3
- HID-HSCT Versus IST as First-line Treatment for SAANot yet recruitingNCT07338422Not applicable
- A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic AnemiaRecruitingNCT05757310Phase 11 country