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ClinicalTrials.gov public records Last synced Oct 16, 2026, 9:21 AM EDT

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Showing 673–696 of 2,014 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Hematological Diseases Clear all
Completed Phase 2 Interventional Results available

Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A

NCT00989196
View directory record Official record
Conditions
Hemophilia A
Interventions
Human-cl rhFVIII, Kogenate FS
Biological
Lead sponsor
Octapharma
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
6
States / cities
Los Angeles, California • Sacramento, California • Denver, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2019 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 2 Interventional

Bryostatin 1 and Cytarabine in Treating Patients With Relapsed Acute Myelogenous Leukemia

NCT00017342
View directory record Official record
Conditions
Leukemia
Interventions
bryostatin 1, cytarabine
Drug
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
4
States / cities
New York, New York • Houston, Texas • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 1, 2010 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 3 Interventional Results available

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

NCT02093897
View directory record Official record
Conditions
Congenital Hemophilia A
Interventions
rVIII-SingleChain
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Aurora, Colorado • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 27, 2017 · Synced Oct 16, 2026, 9:21 AM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT06363760
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2040
U.S. locations
16
States / cities
Oakland, California • Aurora, Colorado • New Haven, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2025 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 2 Interventional

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

NCT00029380
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
Interventions
Sangstat, Cyclophosphamide, Busulfan, Mycophenolate Mofetil, Cyclosporine, Cord Blood Transplantation
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 14 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
14
States / cities
Oakland, California • Washington D.C., District of Columbia • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2016 · Synced Oct 16, 2026, 9:21 AM EDT
Active, not recruiting Not applicable Interventional

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

NCT03047746
View directory record Official record
Conditions
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
Interventions
CliniMACs
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 16, 2026, 9:21 AM EDT
Terminated No phase listed Observational

Change in Thrombin Generation Potential and Thromboelastography During the Menstrual Cycle

NCT00178542
View directory record Official record
Conditions
Von Willebrand Disease
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 6, 2013 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Not applicable Interventional

Individualized Exercise Programs for Patients Receiving Chemotherapy for Newly Diagnosed Acute or Relapsed Myeloid Leukemia or Lymphoblastic Leukemia

NCT00499759
View directory record Official record
Conditions
Depression, Fatigue, Leukemia, Musculoskeletal Complications, Psychosocial Effects of Cancer and Its Treatment
Interventions
Resting vitals, Body Composition, Timed Get-up and Go test (TGUG)., Cardiorespiratory assessment, Handgrip evaluation, Dynamic muscular endurance
Behavioral
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Oct 16, 2026, 9:21 AM 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 16, 2026, 9:21 AM EDT
Completed Phase 2 Interventional Results available

Submucosal Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT01402531
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions
Submucosal Bevacizumab
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Oct 16, 2026, 9:21 AM EDT
Terminated Phase 3 Interventional

A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy

NCT00294541
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
ICA-17043
Drug
Lead sponsor
Icagen
Industry
Eligibility
17 Years to 66 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
31
States / cities
Mobile, Alabama • Little Rock, Arkansas • Oakland, California + 25 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2007 · Synced Oct 16, 2026, 9:21 AM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

NCT02105766
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
Interventions
Alemtuzumab, Sirolimus, Cyclophosphamide, Pentostatin, Radiotherapy, Filgrastim
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A

NCT01493778
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
0 Years to 6 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
25
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2020 · Synced Oct 16, 2026, 9:21 AM EDT
Completed No phase listed Observational

Obesity in Pediatric Sickle Cell Disease: A New Phenomenon

NCT04676113
View directory record Official record
Conditions
Sickle Cell Disease, Obesity, Childhood, Hypertension, Hyperlipidemia in Children, Hyperglycemia
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
10 Years to 19 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated May 12, 2023 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Not applicable Interventional

Use of Mobile Technology for Intensive Training in Medication Management

NCT02133560
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Video recording, Medication Administration Log, Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
5 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 25, 2017 · Synced Oct 16, 2026, 9:21 AM EDT
Completed No phase listed Observational

Liver Fibrosis in Sickle Cell Disease

NCT02007746
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
Other · Procedure
Lead sponsor
University of Miami
Other
Eligibility
10 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Oct 16, 2026, 9:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Microvascular and Fibrosis Imaging Study

NCT01758250
View directory record Official record
Conditions
Autoimmune Diseases, Sickle Cell Disease, Chronic Graft Versus Host Disease
Interventions
Acoustic Radiation Force Impulse (ARFI), Laser Doppler Flowmetry (LDF), Laser Doppler Perfusion Imaging (LDI), Optical Coherence Tomography (OCT), Orthogonal Polarization Spectral Imaging (OPSI), Nail fold video capillaroscopy (NVC)
Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 11, 2020 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 2 Interventional Results available

Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)

NCT00745420
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hematopoietic Stem Cell Transplantation
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
3 Years to 19 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2022 · Synced Oct 16, 2026, 9:21 AM EDT
Terminated Phase 1 Interventional

Donor Stem Cell Transplant in Treating Patients With High Risk Acute Myeloid Leukemia

NCT01801046
View directory record Official record
Conditions
Adult Acute Megakaryoblastic Leukemia (M7), 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 in Remission, 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), Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Monoblastic Leukemia (M5a), Childhood Acute Monocytic Leukemia (M5b), Childhood Acute Myeloblastic Leukemia With Maturation (M2), Childhood Acute Myeloblastic Leukemia Without Maturation (M1), Childhood Acute Myeloid Leukemia in Remission, Childhood Acute Myelomonocytic Leukemia (M4), Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia, Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions
mitoxantrone hydrochloride, cytarabine, peripheral blood stem cell transplantation, laboratory biomarker analysis
Drug · Procedure · Other
Lead sponsor
University of Southern California
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 30, 2018 · Synced Oct 16, 2026, 9:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia

NCT01558076
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
8 Years to 21 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 3 Interventional Results available

Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment

NCT00623480
View directory record Official record
Conditions
Hemophilia A
Interventions
Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Biological
Lead sponsor
Bayer
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
28
States / cities
Tucson, Arizona • Little Rock, Arkansas • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2014 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Phase 2 Interventional Results available

Treatment of POEMS Syndrome With Daratumumab

NCT04396496
View directory record Official record
Conditions
POEMS Syndrome
Interventions
Daratumumab Injection
Drug
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Nov 13, 2023 · Synced Oct 16, 2026, 9:21 AM EDT
Completed Not applicable Interventional Results available

Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant

NCT00293384
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nausea and Vomiting, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
Aprepitant, Cyclophosphamide, Dexamethasone, Granisetron hydrochloride
Drug
Lead sponsor
Barbara Ann Karmanos Cancer Institute
Other
Eligibility
18 Years to 120 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 15, 2016 · Synced Oct 16, 2026, 9:21 AM 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 16, 2026, 9:21 AM EDT