68 recruiting. 68 Phase 1, 35 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 68 | Recruiting 68 |
| Phase 2 | 35 | Recruiting 35 |
Follow Anemia: new trials by email Literature Review of Anemia trials Research Report on Anemia
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 |
|---|---|---|---|---|---|---|
| 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 | 2026-11-04 | 2026-10-30 |
| 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 |
| To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants. Part1: TUL321, Part1: Placebo, Part2: TUL321 - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 1 | Recruiting | Zhuhai United Laboratories Co., Ltd. | 94 | 2026-08-26 | 2026-09-01 |
| Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452 - Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | Phase 1 | Recruiting | Daniel Bauer | 5 | 2026-08-01 | 2026-10-21 |
| Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease VAS-101 - Sickle Cell Disease, Hemolytic Anemia | Phase 1 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 25 | 2026-07-31 | 2026-10-21 |
| A Study of Ferric Derisomaltose Injection in Chinese Participants With Iron Deficiency Anemia Under Fasting Conditions Ferric Derisomaltose Injection, Ferric Derisomaltose Injection [MonoFer®] - Iron Deficiency Anemia | Phase 1 | Recruiting | Sichuan Huiyu Pharmaceutical Co., Ltd | 88 | 2026-07-23 | 2026-10-13 |
| Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors Total Body Irradiation, Alemtuzumab, Abatacept - Sickle Cell Disease, Beta-thalassemia | Phase 1 / Phase 2 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 90 | 2026-07-08 | 2026-10-13 |
| HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy. HY001N Cell Injection - Autoimmune Hemolytic Anemia (AIHA) | Phase 1 | Recruiting | Juventas Cell Therapy Ltd. | 9 | 2026-07-02 | 2026-08-05 |
| 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 |
| Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease Ruxolitinib - Sickle Cell Disease, Hematopoetic Stem Cell Transplant | Phase 1 / Phase 2 | Recruiting | Arkansas Children's Hospital Research Institute | 24 | 2026-06-08 | 2026-06-04 |
| Luspatercept for CIA in AML Luspatercept Injectable Product - Chemotherapy-inducing Anemia | Phase 1 / Phase 2 | Recruiting | Guangdong Second Provincial General Hospital | 40 | 2026-06-01 | 2026-06-23 |
| Therapeutic RSK1 Targeting in Myeloid Malignancies PMD-026 - Myeloid Malignancy | Phase 1 | Recruiting | Washington University School of Medicine | 30 | 2026-05-20 | 2026-10-21 |
| Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study hydroxycarbamide - Sickle Cell Disease, Sickle Cell Anemia in Children | Phase 1 / Phase 2 | Recruiting | Children's Hospital Medical Center, Cincinnati | 250 | 2026-05-13 | 2026-07-16 |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia APR-2020 - RPS19 Deficient Diamond-Blackfan Anemia | Phase 1 | Recruiting | Apriligen, Inc. | 4 | 2026-04-16 | 2026-07-21 |
| A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants CMS-D017 Capsule, CMS-D017 Placebo Capsule - Paroxysmal Nocturnal Hemoglobinuria, PNH, Complement-mediated Kidney Disease | Phase 1 | Recruiting | Shenzhen Kangzhe Biotechnology Co., Ltd. | 88 | 2026-03-05 | 2026-04-08 |
| Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia zoledronic acid - Congenital Dyserythropoietic Anemia (CDA) | Phase 1 / Phase 2 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 2 | 2026-02-25 | 2026-03-13 |
| Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA. DREAM01 drug product, anti-inflammatory therapy - Sickle Cell Disease | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 15 | 2026-02-25 | 2026-03-04 |
| Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria HS-10542 - Paroxysmal Nocturnal Hemoglobinuria | Phase 1 / Phase 2 | Recruiting | Jiangsu Hansoh Pharmaceutical Co., Ltd. | 50 | 2026-02-14 | 2026-04-13 |
| A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor ALN-CFB, Placebo - Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia | Phase 1 / Phase 2 | Recruiting | Regeneron Pharmaceuticals | 24 | 2026-02-11 | 2026-07-09 |
| A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) DISC-3405 - Sickle Cell Disease | Phase 1 | Recruiting | Disc Medicine, Inc | 24 | 2026-01-06 | 2026-10-05 |
Showing 20 of 68. Sign in to see all of them.
Anemia, Sickle Cell (24) · Anemia, Refractory, with Excess of Blasts (10) · Leukemia, Myeloid, Acute (10) · Hemoglobinuria, Paroxysmal (7) · Anemia, Aplastic (5) · Myelodysplastic Syndromes (5) · beta-Thalassemia (5) · Anemia, Hemolytic, Autoimmune (4) · Fanconi Anemia (3) · Hemoglobinopathies (3) · Leukemia, Myelomonocytic, Chronic (3) · Myeloproliferative Disorders (3) · Primary Myelofibrosis (3) · Anemia, Diamond-Blackfan (2)
Hemic and Lymphatic Diseases (68) · Hematologic Diseases (66) · Anemia, Hemolytic (37) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (35) · Genetic Diseases, Inborn (34) · Bone Marrow Diseases (29) · Anemia, Hemolytic, Congenital (27) · Hemoglobinopathies (25) · Myelodysplastic Syndromes (15) · Neoplasms (14) · Neoplasms by Histologic Type (14) · Leukemia, Myeloid (11) · Anemia, Refractory (10) · Bone Marrow Failure Disorders (10)
Cyclophosphamide (4) · Whole-Body Irradiation (4) · Alemtuzumab (3) · fludarabine (3) · fludarabine phosphate (3) · Abatacept (2) · Biopsy (2) · Busulfan (2) · Hydroxyurea (2) · Mycophenolic Acid (2) · Peripheral Blood Stem Cell Transplantation (2) · Rituximab (2) · Sequence Analysis (2) · Sirolimus (2) · 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene (1)
Institute of Hematology & Blood Diseases Hospital, China (4) · National Heart, Lung, and Blood Institute (NHLBI) (4) · St. Jude Children's Research Hospital (3) · Daniel Bauer (2) · Disc Medicine, Inc (2) · Fred Hutchinson Cancer Center (2) · Washington University School of Medicine (2) · ADARx Pharmaceuticals, Inc. (1) · ARCE Therapeutics, Inc. (1) · Abdullah Kutlar (1) · Apollo Therapeutics Ltd (1) · Apriligen, Inc. (1) · Arkansas Children's Hospital Research Institute (1) · Ascentage Pharma Group Inc. (1) · Assistance Publique - Hôpitaux de Paris (1)
United States (35) · China (23) · Australia (4) · United Kingdom (4) · Canada (3) · France (3) · Brazil (1) · Czechia (1) · Germany (1) · Israel (1) · Italy (1) · South Korea (1) · Spain (1) · Turkey (Türkiye) (1) · Uganda (1)
Swee Lay Thein, M.D. (2) · Abdullah Kutlar, MD (1) · Agata Smogorzewska, MD, PhD (1) · Akshay Sharma, MBBS, MSc (1) · Alexis Leonard, MD (1) · Amy W Zhou, M.D. (1) · Andrew W. Heitzer, PhD (1) · Angela Flesichman, MD, PhD (1) · Brenda M. Sandmaier (1) · Chao Liu, PHD (1) · Claudia Morris, MD (1) · Fengkui Zhang, Dr. (1)
PMID 20236457, PMID 21325598, PMID 23594433, PMID 24300013, PMID 24691607, PMID 25805204, PMID 26443879, PMID 26836806, PMID 28131429, PMID 30181084, PMID 30335787, PMID 30444173, PMID 30860564, PMID 31723612, PMID 32384147, PMID 32813873, PMID 33475735, PMID 38527291, PMID 39722321, PMID 40696207, PMID 40859225, PMID 42345101, PMID 42549972, PMID 9971864
P01CA078902 (2), 2023 (1), 2025 (1), K24AT009893 (1), P30CA015704 (1), P30CA033572 (1), U01HL163983 (1)