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ClinicalTrials.gov public records Last synced Oct 15, 2026, 4:08 PM EDT

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Showing 505–528 of 2,014 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Hematological Diseases Clear all
Completed Phase 2 Interventional Results available

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

NCT00053989
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Fanconi Anemia, Aplastic Anemia
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, therapeutic allogeneic lymphocytes, cyclophosphamide, fludarabine phosphate, methylprednisolone, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 75 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2018
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Feb 10, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Not yet recruiting No phase listed Observational

Clinical Genetics Branch Eligibility Screening Survey

NCT07005297
View directory record Official record
Conditions
Melanoma, Li-Fraumeni Syndrome, Pulmonary Blastoma, Chordoma, Congenital Bone Marrow Failure Syndromes, Costello Syndrome, Fanconi Anemia, CFC Syndrome (CFCS), Legius Syndrome, RASopathies
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Not applicable Interventional Results available

Clinical Trial of Factor XIII (FXIII) Concentrate

NCT00640289
View directory record Official record
Conditions
Hemophilia, Factor XIII Deficiency
Interventions
Fibrogammin P
Drug
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
Not listed
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Nov 7, 2018 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 3 Interventional Results available

Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally

NCT01380197
View directory record Official record
Conditions
Pain, Sickle Cell Disease
Interventions
Morphine, Nubain
Drug
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
6 Years to 19 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 20, 2018 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management

NCT03291613
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pinpoint App
Other
Lead sponsor
Klein Buendel, Inc.
Industry
Eligibility
13 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017
U.S. locations
2
States / cities
Golden, Colorado • Munster, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 6, 2019 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 3 Interventional Results available

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

NCT01440946
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIXFc, FIX
Drug
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
7
States / cities
Phoenix, Arizona • Sacramento, California • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 2 Interventional

Bone Marrow Transplant in Treating Patients With Hematologic Cancers

NCT00005797
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
busulfan, Cyclophosphamide, VP-16, Fractionated Total Body Irradiation (FTBI)
Drug · Radiation
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
15 Years to 55 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2007
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Dec 10, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Imatinib for Pain in Sickle Cell Anemia

NCT03997903
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Imatinib Mesylate
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 25 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
2
States / cities
Indianapolis, Indiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Oct 15, 2026, 4:08 PM EDT
Terminated Phase 1 Interventional

Single Agent and Combined Inhibition After Allogeneic Stem Cell Transplant

NCT02846376
View directory record Official record
Conditions
Acute Myeloid Leukemia and Myelodysplastic Syndrome
Interventions
Nivolumab, Ipilimumab
Drug
Lead sponsor
Hackensack Meridian Health
Other
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Hackensack, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 18, 2024 · Synced Oct 15, 2026, 4:08 PM EDT
Completed No phase listed Observational

Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)

NCT02466789
View directory record Official record
Conditions
Von Willebrands Disease
Interventions
Not listed
Lead sponsor
Jonathan Roberts
Other
Eligibility
2 Years and older
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
1
States / cities
Peoria, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 10, 2022 · Synced Oct 15, 2026, 4:08 PM EDT
Completed No phase listed Observational

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

NCT02368340
View directory record Official record
Conditions
Hermansky Pudlak Syndrome
Interventions
Pulmonary function test, Chest CT, Sample collection
Other
Lead sponsor
Vanderbilt University
Other
Eligibility
12 Years to 90 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 1 Interventional

MS-275 in Treating Patients With Hematologic Cancer

NCT00015925
View directory record Official record
Conditions
Leukemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
entinostat
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2010 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 4 Interventional

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

NCT00092976
View directory record Official record
Conditions
Hemophilia A
Interventions
ReFacto
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
6 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
4
States / cities
Los Angeles, California • New Brunswick, New Jersey • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2007 · Synced Oct 15, 2026, 4:08 PM EDT
Completed No phase listed Observational Results available

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

NCT01783990
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
24 Months to 18 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Completed No phase listed Observational

Genetic Studies of X-linked Lymphoproliferative Disease

NCT00359411
View directory record Official record
Conditions
X-Linked Lymphoproliferative Disease, Lymphoproliferative Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 4:08 PM EDT
Terminated Phase 2 Interventional Results available

Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer

NCT00818961
View directory record Official record
Conditions
Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
alemtuzumab, graft-versus-tumor induction therapy, rituximab, busulfan, cyclophosphamide, fludarabine phosphate, methotrexate, tacrolimus, allogeneic bone marrow transplantation
Biological · Drug · Procedure
Lead sponsor
Northside Hospital, Inc.
Other
Eligibility
40 Years to 72 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 18, 2013 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 3 Interventional Results available

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

NCT03938792
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
PF-06741086
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years to 74 Years · Male only
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
4
States / cities
Tampa, Florida • Iowa City, Iowa • New Hyde Park, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 15, 2026, 4:08 PM EDT
Not listed Phase 2 Interventional

Combination Chemotherapy Followed by Donor Bone Marrow Transplant or Peripheral Stem Cell Transplant in Treating Patients With Hematologic Cancer or Genetic Disorders

NCT00008307
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, cyclosporine, fludarabine phosphate, melphalan, methylprednisolone, mycophenolate mofetil, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, syngeneic bone marrow transplantation
Biological · Drug · Procedure
Lead sponsor
Herbert Irving Comprehensive Cancer Center
Other
Eligibility
1 Year to 80 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2014 · Synced Oct 15, 2026, 4:08 PM EDT
Terminated No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 15, 2026, 4:08 PM 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 15, 2026, 4:08 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers Results available

Sickle Cell Trait and the Risk of Venous Thromboembolism

NCT01148940
View directory record Official record
Conditions
Thrombosis
Interventions
Not listed
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Dec 17, 2020 · Synced Oct 15, 2026, 4:08 PM EDT
Completed No phase listed Observational

Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging

NCT03478917
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
10 Years to 85 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 17, 2023 · Synced Oct 15, 2026, 4:08 PM EDT
Completed Phase 1 Interventional Results available

A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease

NCT02314689
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Intravenous (IV) citrulline
Drug
Lead sponsor
Suvankar Majumdar
Other
Eligibility
18 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Sep 3, 2019 · Synced Oct 15, 2026, 4:08 PM EDT
Terminated Phase 3 Interventional Results available

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

NCT01271868
View directory record Official record
Conditions
Hemophilia B
Interventions
IB1001
Biological
Lead sponsor
Medexus Pharma, Inc.
Industry
Eligibility
Up to 12 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Indianapolis, Indiana • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced Oct 15, 2026, 4:08 PM EDT