Bone Marrow Diseases: Clinical Trials

431 trials for Bone Marrow Diseases, newest first

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232 completed, 54 terminated, 48 unknown, 38 active not recruiting. 431 Phase 3, 62 Phase 2.

By phase and status

PhaseTrialsStatus
Phase 3431Completed 232, Terminated 54, Unknown 48, Active not recruiting 38, Recruiting 31, Not yet recruiting 19, Withdrawn 6, Suspended 2, Enrolling by invitation 1
Phase 262Completed 28, Unknown 15, Recruiting 5, Terminated 5, Not yet recruiting 4, Active not recruiting 3, Withdrawn 2

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Trials (filtered) newest first · largest first

All 431 trials

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

TrialPhaseStatusSponsorEnrollmentStartUpdated
Sonrotoclax in Combination With Azacitidine for Previously Untreated Higher-risk Myelodysplastic Syndromes
Each treatment cycle is 28-days. Azacitidine 75 mg/m² is administered subcutaneously or intravenously on Days 1-7 of each cycle. Sonrotoclax is orally administered once daily on Days 1-14 within each - MDS (Myelodysplastic Syndrome)
Phase 2 / Phase 3Not yet recruitingThe Affiliated People's Hospital of Ningbo University102028-09-012026-11-12
Methadone as an Alternative Treatment for Children Underdoing HSCT
PCA plus methadone (Experimental), PCA Alone (SOC) - Leukemia, Aplastic Anemia
Phase 3SuspendedUniversity of Texas Southwestern Medical Center602028-01-012025-10-23
A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS)
Roxadustat, Placebo - Myelodysplastic Syndromes
Phase 3Not yet recruitingKyntra Bio2012027-04-012026-07-23
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
FXS6837 Capsule - Paroxysmal Nocturnal Hemoglobinuria
Phase 3Not yet recruitingShanghai Fosun Pharmaceutical Industrial Development Co. Ltd.202027-01-262026-11-04
A Randomized Study of ELVN-001 Versus Investigator-Selected TKI, in CP-CML Previously Treated With One or More TKIs
ELVN-001, TKI - Chronic Myeloid Leukemia (CML), Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive
Phase 3Not yet recruitingEnliven Therapeutics4502026-12-312026-10-29
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
FXS6837 Capsule, Eculizumab infusion - Paroxysmal Nocturnal Hemoglobinuria
Phase 3Not yet recruitingShanghai Fosun Pharmaceutical Industrial Development Co. Ltd.902026-12-102026-08-05
A Study of HS-10382 Versus Investigator-selected TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP
HS-10382, Imatinib, Dasatinib - Chronic Myeloid Leukemia
Phase 3Not yet recruitingJiangsu Hansoh Pharmaceutical Co., Ltd.4082026-12-012026-10-21
A Study to Investigate AG-236 in Subjects ≥18 Years of Age With Polycythemia Vera
AG-236, Placebo - Polycythemia Vera
Phase 2 / Phase 3Not yet recruitingAgios Pharmaceuticals, Inc.2452026-11-012026-10-13
Efficacy and Safety of Maximum Labeled Dose Luspatercept in Lower-Risk Myelodysplastic Syndromes
Luspatercept - Myelodysplastic Syndromes
Phase 3Not yet recruitingInstitute of Hematology & Blood Diseases Hospital, China752026-10-312026-10-26
A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
VSA012, Eculizumab (Soliris®) - Paroxysmal Nocturnal Hemoglobinuria, PNH
Phase 3Not yet recruitingBisirna Therapeutics (Suzhou) Co., Ltd.702026-10-292026-09-11
A Clinical Trial of Bomedemstat in Participants With Polycythemia Vera (MK-3543-025).
Bomedemstat, Ruxolitinib, Ropeginterferon alfa-2b - Polycythemia Vera
Phase 2 / Phase 3RecruitingMerck Sharp & Dohme LLC3802026-10-292026-11-05
Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients
N-acetylcysteine - Aplastic Anemia
Phase 3Not yet recruitingPeking University People's Hospital1422026-09-302026-09-16
A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
Placebo, Elritercept - Myelofibrosis, Anemia
Phase 3RecruitingTakeda3242026-09-102026-11-06
A Phase 3 Study of INCA033989 Versus Best Available Therapy in Participants With Essential Thrombocythemia
INCA033989, Best Available Treatment - Essential Thrombocythemia
Phase 3RecruitingIncyte Corporation4262026-09-082026-11-05
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 3Not yet recruitingSwedish Orphan Biovitrum262026-07-312026-08-26
Mitoxantrone Hydrochloride Liposome, Cytarabine, G-CSF Plus Venetoclax vs. Azacitidine Plus Venetoclax for MDS-IB2 and Secondary/Elderly AML
Mitoxantrone Hydrochloride Liposome, Cytarabine, Granulocyte Colony-Stimulating Factor(G-CSF) - Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
Phase 3Not yet recruitingRuijin Hospital1682026-07-302026-07-29
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria
Phase 3RecruitingRegeneron Pharmaceuticals352026-06-112026-08-05
A Long-term Efficacy and Safety of NTQ5082 Capsules
NTQ5082 capsules 200 mg - Paroxysmal Nocturnal Hemoglobinuria
Phase 3Not yet recruitingNanjing Chia-tai Tianqing Pharmaceutical782026-06-012025-09-17
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Pelabresib, Ruxolitinib, Placebo - Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
Phase 3RecruitingNovartis Pharmaceuticals4602026-05-272026-11-10
INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
Sapablursen, Placebo - Polycythemia Vera
Phase 3RecruitingOno Pharmaceutical Co., Ltd.2502026-05-252026-10-29

Showing 20 of 431. Sign in to see all of them.

Narrower conditions

Myelodysplastic Syndromes (151)  ·  Leukemia, Myeloid, Acute (106)  ·  Leukemia (92)  ·  Leukemia, Myelogenous, Chronic, BCR-ABL Positive (85)  ·  Myeloproliferative Disorders (65)  ·  Precursor Cell Lymphoblastic Leukemia-Lymphoma (64)  ·  Leukemia, Myeloid, Chronic-Phase (58)  ·  Primary Myelofibrosis (58)  ·  Hemoglobinuria, Paroxysmal (50)  ·  Leukemia, Myelomonocytic, Chronic (45)  ·  Lymphoma (36)  ·  Polycythemia Vera (35)  ·  Lymphoma, Non-Hodgkin (34)  ·  Anemia (32)  ·  Leukemia, Myeloid, Accelerated Phase (32)

Broader conditions

Hemic and Lymphatic Diseases (431)  ·  Hematologic Diseases (414)  ·  Neoplasms (240)  ·  Neoplasms by Histologic Type (226)  ·  Leukemia, Myeloid (204)  ·  Pathological Conditions, Signs and Symptoms (176)  ·  Myeloproliferative Disorders (168)  ·  Pathologic Processes (168)  ·  Disease Attributes (159)  ·  Chronic Disease (153)  ·  Leukemia (135)  ·  Anemia (105)  ·  Immune System Diseases (89)  ·  Immunoproliferative Disorders (80)

Interventions

Cytarabine (41)  ·  Cyclophosphamide (40)  ·  Imatinib Mesylate (34)  ·  Busulfan (32)  ·  nilotinib (26)  ·  Etoposide (24)  ·  eculizumab (24)  ·  ruxolitinib (23)  ·  Methotrexate (22)  ·  Azacitidine (21)  ·  Daunorubicin (21)  ·  fludarabine phosphate (21)  ·  Hydroxyurea (20)  ·  Peripheral Blood Stem Cell Transplantation (20)  ·  Filgrastim (18)

Sponsors

Novartis Pharmaceuticals (37)  ·  Alexion Pharmaceuticals, Inc. (12)  ·  European Organisation for Research and Treatment of Cancer - EORTC (12)  ·  Celgene (11)  ·  Bristol-Myers Squibb (8)  ·  Fred Hutchinson Cancer Center (8)  ·  M.D. Anderson Cancer Center (8)  ·  AOP Orphan Pharmaceuticals AG (7)  ·  Merck Sharp & Dohme LLC (7)  ·  Alliance for Clinical Trials in Oncology (6)  ·  Children's Oncology Group (6)  ·  Incyte Corporation (6)  ·  Medical College of Wisconsin (6)  ·  National Cancer Institute (NCI) (6)  ·  Amgen (5)

Countries

United States (209)  ·  France (135)  ·  Germany (134)  ·  Italy (130)  ·  United Kingdom (118)  ·  Spain (109)  ·  Canada (105)  ·  Belgium (86)  ·  Australia (80)  ·  Poland (78)  ·  Czechia (75)  ·  Netherlands (75)  ·  South Korea (72)  ·  China (68)  ·  Hungary (65)

Top investigators

Mary Horowitz, MD (5)  ·  Qifa Liu (4)  ·  Alan K. Burnett, MD, FRCP (3)  ·  Heinz Gisslinger, MD (3)  ·  Ruediger Hehlmann, MD (3)

Results publications

PMID 31375516, PMID 36184192, PMID 37042865, PMID 39516087, PMID 39535306, PMID 40535755, PMID 40694294, PMID 41958363, PMID 42316774, PMID 23515925, PMID 23620574, PMID 23911705, PMID 24859364, PMID 26069290, PMID 28273028, PMID 28962635, PMID 29396713, PMID 29926156, PMID 30523055, PMID 32499241, PMID 33275766, PMID 33811823, PMID 34526067, PMID 35235189, PMID 36815378, PMID 37267439, PMID 37311468, PMID 37341189, PMID 37553873, PMID 38065202, PMID 38295280, PMID 38477987, PMID 38727860, PMID 39079163, PMID 40447351, PMID 40720060, PMID 40978458, PMID 41890273, PMID 42085603, PMID 42452788, PMID 12637609, PMID 15010373, PMID 15459300, PMID 15761020, PMID 16136166, PMID 16333838, PMID 16484584, PMID 16932345, PMID 16960978, PMID 16990386

NIH funding

2023 (17), U01HL069294 (6), 2009 (5), 2015 (5), 2018 (5), 2019 (5), U10CA021115 (5), 2014 (4), 2016 (4), 2017 (4), U24CA076518 (4), 2025 (3), U10CA031946 (3), 2004 (2), 2005 (2)