Anemia, Aplastic: Clinical Trials

211 completed, 72 unknown, 64 recruiting, 42 terminated. 232 Phase 2, 98 Phase 1, 56 Not applicable, 31 Phase 3, 20 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	232	Completed 110, Unknown 32, Recruiting 30, Terminated 23, Not yet recruiting 16, Active not recruiting 12, Withdrawn 8, Enrolling by invitation 1
Phase 1	98	Completed 42, Unknown 15, Terminated 14, Recruiting 10, Not yet recruiting 7, Active not recruiting 6, Withdrawn 3, Enrolling by invitation 1
Not applicable	56	Completed 24, Unknown 11, Terminated 6, Withdrawn 6, Recruiting 5, Active not recruiting 2, Not yet recruiting 2
Phase 3	31	Completed 17, Unknown 5, Not yet recruiting 4, Terminated 3, Suspended 1, Withdrawn 1
Phase 4	20	Unknown 11, Completed 7, Terminated 1, Withdrawn 1
Early Phase 1	5	Completed 2, Recruiting 2, Terminated 1

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Methadone as an Alternative Treatment for Children Underdoing HSCT PCA plus methadone (Experimental), PCA Alone (SOC) - Leukemia, Aplastic Anemia	Phase 3	Suspended	University of Texas Southwestern Medical Center	60	2028-01-01	2025-10-23
Prenatal Transplantation for Fetuses With Fanconi Anemia IUHSCT for FA-affected fetuses - Fanconi Anemia, Anemia, Hypoplastic, Congenital	Phase 1 / Phase 2	Not yet recruiting	Agnieszka Czechowicz	12	2028-01-01	2026-06-16
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) - Aplastic Anaemia	Phase 2	Not yet recruiting	Anhui Provincial Hospital	59	2027-08-01	2026-10-21
CK0801 for Treatment of Aplastic Anemia CK0801 - Aplastic Anemia	Phase 2	Not yet recruiting	Cellenkos, Inc.	12	2026-12-03	2026-10-20
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 pacritinib - Myelodysplastic Syndromes	Phase 1 / Phase 2	Recruiting	National Cancer Institute (NCI)	160	2026-11-03	2026-10-29
Clinical Genetics Branch Eligibility Screening Survey Melanoma, Li-Fraumeni Syndrome		Not yet recruiting	National Cancer Institute (NCI)	1000	2026-11-03	2026-10-29
Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia Metformin, Naproxen Sodium - Fanconi Anemia, Head and Neck Cancer	Phase 1	Recruiting	Rockefeller University	80	2026-10-14	2026-10-28
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI Sirolimus, Ruxolitinib, MMF - Acute Leukemia, Myelodysplastic Syndromes	Phase 1 / Phase 2	Not yet recruiting	Peking University People's Hospital	40	2026-10-01	2026-09-09
Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients N-acetylcysteine - Aplastic Anemia	Phase 3	Not yet recruiting	Peking University People's Hospital	142	2026-09-30	2026-09-16
Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic Anemia Thiotepa - Severe Aplastic Anemia, Hematopoietic Cell Transplant		Not yet recruiting	Peking University People's Hospital	20	2026-09-20	2026-09-21
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes Dibotatug (DR-01) - Bone Marrow Failure Syndromes, Aplastic Anemias	Phase 1 / Phase 2	Not yet recruiting	Dren Bio	60	2026-08-01	2026-07-24
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment Avatrombopag 20 mg Oral Tablet - Aplastic Anemia	Phase 2 / Phase 3	Not yet recruiting	Swedish Orphan Biovitrum	26	2026-07-31	2026-08-26
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells - Leukemia, Lymphoma	Phase 2	Not yet recruiting	Hematology department of the 920th hospital	60	2026-07-03	2026-07-14
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia Isatuximab - Aplastic Anemia	Phase 1 / Phase 2	Recruiting	Institute of Hematology & Blood Diseases Hospital, China	32	2026-06-30	2026-09-21
MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients MRIs - Fanconi Anemia		Recruiting	University of Minnesota	80	2026-06-23	2026-07-02
Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia G-CSF-Primed Peripheral Blood Stem Cell Graft, G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft - Severe Aplastic Anemia (SAA)	Not applicable	Recruiting	Peking University People's Hospital	180	2026-06-15	2026-09-16
Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA Romiplostim N01 - Aplastic Anemia	Phase 2	Withdrawn	Peking Union Medical College Hospital		2026-06-01	2026-06-04
A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA BFCA conditiong regimen - Severe Aplastic Anemia, Aplastic Anaemia		Recruiting	Peking University People's Hospital	64	2026-06-01	2026-07-06
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector Gene-modified autologous stem cells - Fanconi Anemia	Not applicable	Terminated	Shenzhen Geno-Immune Medical Institute	10	2026-06-01	2026-08-24
CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01 - Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia	Phase 2	Withdrawn	Peking Union Medical College Hospital		2026-06-01	2026-06-04

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Narrower conditions

Fanconi Anemia (95)  ·  Myelodysplastic Syndromes (95)  ·  Anemia, Diamond-Blackfan (56)  ·  Leukemia, Myeloid, Acute (56)  ·  Precursor Cell Lymphoblastic Leukemia-Lymphoma (50)  ·  Multiple Myeloma (47)  ·  Bone Marrow Failure Disorders (46)  ·  Lymphoma, Non-Hodgkin (46)  ·  Leukemia (45)  ·  Hodgkin Disease (44)  ·  Anemia, Sickle Cell (38)  ·  Leukemia, Lymphocytic, Chronic, B-Cell (38)  ·  Lymphoma (36)  ·  Myeloproliferative Disorders (32)

Broader conditions

Hemic and Lymphatic Diseases (460)  ·  Bone Marrow Diseases (458)  ·  Anemia (444)  ·  Hematologic Diseases (435)  ·  Bone Marrow Failure Disorders (415)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (172)  ·  Genetic Diseases, Inborn (153)  ·  Anemia, Hypoplastic, Congenital (133)  ·  Congenital Bone Marrow Failure Syndromes (132)  ·  Nutritional and Metabolic Diseases (130)  ·  Immune System Diseases (125)  ·  Metabolic Diseases (122)  ·  Pathological Conditions, Signs and Symptoms (104)  ·  Neoplasms by Histologic Type (101)

Interventions

Cyclophosphamide (79)  ·  fludarabine (59)  ·  Cyclosporine (58)  ·  Antilymphocyte Serum (55)  ·  fludarabine phosphate (46)  ·  Whole-Body Irradiation (40)  ·  thymoglobulin (35)  ·  Busulfan (30)  ·  Alemtuzumab (27)  ·  Mycophenolic Acid (27)  ·  Melphalan (24)  ·  Tacrolimus (24)  ·  eltrombopag (24)  ·  Filgrastim (22)  ·  Granulocyte Colony-Stimulating Factor (21)

Sponsors

National Heart, Lung, and Blood Institute (NHLBI) (46)  ·  Institute of Hematology & Blood Diseases Hospital, China (27)  ·  Peking Union Medical College Hospital (19)  ·  Novartis Pharmaceuticals (18)  ·  Masonic Cancer Center, University of Minnesota (13)  ·  Fred Hutchinson Cancer Center (11)  ·  Peking University People's Hospital (10)  ·  M.D. Anderson Cancer Center (9)  ·  Assistance Publique - Hôpitaux de Paris (8)  ·  University of California, San Francisco (8)  ·  Children's Hospital Medical Center, Cincinnati (7)  ·  Roswell Park Cancer Institute (7)  ·  City of Hope Medical Center (6)  ·  Memorial Sloan Kettering Cancer Center (6)  ·  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (6)

Countries

United States (252)  ·  China (83)  ·  United Kingdom (18)  ·  South Korea (16)  ·  France (15)  ·  Italy (15)  ·  Spain (15)  ·  Germany (12)  ·  Japan (11)  ·  Switzerland (7)  ·  Belgium (6)  ·  Brazil (6)  ·  Netherlands (6)  ·  Russia (6)  ·  Australia (5)

Top investigators

Neal S Young, M.D. (8)  ·  Danielle M Townsley, M.D. (5)  ·  Bhavisha A Patel, M.D. (4)  ·  Richard W Childs, M.D. (4)  ·  Adrianna Vlachos, MD (3)  ·  Bing Bing, PhD (3)  ·  Cynthia E Dunbar, M.D. (3)  ·  Farid Boulad, MD (3)  ·  Hans-Peter Kiem (3)  ·  Joanne Kurtzberg, MD (3)  ·  Margaret MacMillan, MD (3)  ·  Paul Szabolcs, MD (3)  ·  Usama Gergis, MD (3)

Results publications

PMID 34724566, PMID 29958797, PMID 29674506, PMID 30504345, PMID 1090310, PMID 18379007, PMID 20685256, PMID 21233311, PMID 22055111, PMID 22067384, PMID 22762314, PMID 25310303, PMID 25824692, PMID 28423296, PMID 30992268, PMID 32343796, PMID 35371427, PMID 39642902, PMID 40593138, PMID 12622583, PMID 18723429, PMID 19117511, PMID 19181786, PMID 19951979, PMID 19996412, PMID 20383216, PMID 20566896, PMID 21071497, PMID 21606164, PMID 21663651, PMID 21715313, PMID 21742281, PMID 21874061, PMID 22517900, PMID 22526363, PMID 22544699, PMID 22546497, PMID 22766223, PMID 22796534, PMID 23585526, PMID 23731760, PMID 24296492, PMID 24319166, PMID 24319167, PMID 24345753, PMID 24370862, PMID 24385534, PMID 24415649, PMID 24786393, PMID 24838091

NIH funding

P30CA015704 (4), P30CA033572 (4), R01HL081499 (3), 2004 (2), 2014 (2), R01DK057778 (2), U10HL069294 (2), U24CA076518 (2), 1UG3HL157564-01A1 (1), 2015 (1), 2016 (1), 2019 (1), 2023 (1), K12CA167540 (1), P01HL036444 (1)