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ClinicalTrials.gov public records Last synced Oct 14, 2026, 7:46 PM EDT

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Showing 265–288 of 2,013 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Hematological Diseases Clear all
Completed No phase listed Observational

Platelet Function in Patients With Hemophilia A

NCT02093065
View directory record Official record
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 18 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 14, 2020 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional Results available

Vitamin D for Sickle-cell Respiratory Complications

NCT01443728
View directory record Official record
Conditions
Sickle Cell Disease, Vitamin D Deficiency, Acute Chest Syndrome, Asthma, Respiratory Infections
Interventions
Experimental: Vitamin D3 100,000 IU, Active Comparator: Vitamin D3 12,000 IU
Drug
Lead sponsor
Gary M Brittenham, MD
Other
Eligibility
3 Years to 20 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 14, 2026, 7:46 PM EDT
Active, not recruiting Phase 1 Interventional

A Blood Stem Cell Transplant for Sickle Cell Disease

NCT03249831
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
Interventions
Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin, Tacrolimus, Mycophenolate mofetil, CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
Drug · Biological
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 45 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional Results available

Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease

NCT02061202
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Mometasone Furoate, Placebo
Drug
Lead sponsor
Jeffrey Glassberg
Other
Eligibility
15 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Oct 14, 2026, 7:46 PM EDT
Terminated Phase 1 Interventional

Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease

NCT02012777
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
propranolol
Drug
Lead sponsor
University of Miami
Other
Eligibility
10 Years to 17 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 8, 2017 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional Results available

Alvocidib, Cytarabine, and Mitoxantrone in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

NCT00795002
View directory record Official record
Conditions
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
alvocidib, mitoxantrone hydrochloride, cytarabine, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
2
States / cities
Baltimore, Maryland • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 7, 2018 · Synced Oct 14, 2026, 7:46 PM EDT
Recruiting Phase 2 Interventional

Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)

NCT07136857
View directory record Official record
Conditions
Glanzmann Thrombasthenia
Interventions
EPTACOG BETA
Drug
Lead sponsor
Emory University
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Oct 14, 2026, 7:46 PM EDT
Recruiting Not applicable Interventional

A Socio-ecological Approach for Improving Self-management in Adolescents With SCD

NCT06290401
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
SCThrive, SCHealthED
Behavioral · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
13 Years to 21 Years
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
4
States / cities
Wilmington, Delaware • Atlanta, Georgia • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional

Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies

NCT00038207
View directory record Official record
Conditions
Soft Tissue Sarcoma, Lymphoma, Leukemia, Wilms' Tumor, Osteosarcoma
Interventions
Liposomal Vincristine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 31, 2018 · Synced Oct 14, 2026, 7:46 PM EDT
Completed No phase listed Observational

Disseminating NIH Evidence Based Sickle Cell Recommendations in North Carolina

NCT03253211
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
4,392 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Raleigh, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 27, 2020 · Synced Oct 14, 2026, 7:46 PM EDT
Terminated Phase 1Phase 2 Interventional

Therapeutic Allogeneic Lymphocytes and Aldesleukin in Treating Patients With High-Risk or Recurrent Myeloid Leukemia After Undergoing Donor Stem Cell Transplant

NCT00052598
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia
Interventions
therapeutic allogeneic lymphocytes, aldesleukin, laboratory biomarker analysis, flow cytometry
Biological · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Feb 15, 2017 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional Results available

Glutamine in Treating Neuropathy Caused by Vincristine in Young Patients With Lymphoma, Leukemia, or Solid Tumors

NCT00365768
View directory record Official record
Conditions
Kidney Cancer, Leukemia, Lymphoma, Neurotoxicity, Peripheral Neuropathy, Sarcoma
Interventions
Glutamine, Placebo
Drug · Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years to 21 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 14, 2026, 7:46 PM EDT
Completed No phase listed Observational

Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia

NCT03185897
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Non-treatment, seroprevalence
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
242 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • East Lansing, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2021 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 2 Interventional Results available

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

NCT03263559
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, Mesna
Procedure · Drug · Radiation
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
5 Years to 45 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
32
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 14, 2026, 7:46 PM EDT
Recruiting No phase listed Observational

Impact of IV Iron on Bleeding Symptoms in Iron Deficient Patients With Inherited Bleeding Disorders

NCT07083583
View directory record Official record
Conditions
Bleeding Disorders, Iron, Platelet, Bleeding
Interventions
IV Iron (standard of care)
Drug
Lead sponsor
Nicoletta C Machin
Other
Eligibility
15 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 14, 2026, 7:46 PM EDT
Not yet recruiting Phase 2 Interventional

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

NCT07660783
View directory record Official record
Conditions
Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia, Immune Dysregulation, Hemophagocytic Lymphohistiocytosis (HLH), Non Malignant Disorders
Interventions
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
6 Months to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 14, 2026, 7:46 PM EDT
Completed No phase listed Observational

GAS-Hem Feasibility Study

NCT02674997
View directory record Official record
Conditions
Hemophilia A
Interventions
Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
5 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
2
States / cities
Peoria, Illinois • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 3 Interventional Results available

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

NCT01480180
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
186 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
31
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 26 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A

NCT03020160
View directory record Official record
Conditions
Hemophilia A
Interventions
Emicizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Sacramento, California • Indianapolis, Indiana • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2023 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Not applicable Interventional

Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell Disease

NCT02857023
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Cogmed RM
Behavioral
Lead sponsor
Steven J. Hardy
Other
Eligibility
7 Years to 16 Years
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Nov 23, 2018 · Synced Oct 14, 2026, 7:46 PM EDT
Terminated Phase 2 Interventional Results available

Treatment of Hemoglobin SC Disease With Hydroxyurea

NCT02336373
View directory record Official record
Conditions
Hemoglobin SC Disease
Interventions
hydroxyurea
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
5 Years to 21 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 9, 2020 · Synced Oct 14, 2026, 7:46 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

NCT01294891
View directory record Official record
Conditions
Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Imaging
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
19 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Oct 14, 2026, 7:46 PM EDT
Terminated Phase 1 Interventional

Donor T Cells in Treating Patients With High-Risk Hematologic Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant

NCT00725062
View directory record Official record
Conditions
Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
CD4+CD25+ regulatory T cells, allogeneic hematopoietic stem cell transplantation
Biological · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years to 70 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 29, 2017 · Synced Oct 14, 2026, 7:46 PM EDT
Completed Phase 1Phase 2 Interventional

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

NCT04684940
View directory record Official record
Conditions
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Interventions
Valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 14, 2026, 7:46 PM EDT