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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

NCT01044186
View directory record Official record
Conditions
Transfusional Iron Overload
Interventions
ICL670
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
8
States / cities
Oakland, California • Orange, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2017 · Synced Sep 28, 2026, 3:15 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
BEAM-101
Biological
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
19
States / cities
Birmingham, Alabama • Phoenix, Arizona • Jacksonville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
lovo-cel
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 2 Interventional Results available

Intravaneous Iron(1000 mg Low Molecular Weight Iron Dextran Over 60 Minutes) for Pregnant Women

NCT02038023
View directory record Official record
Conditions
Pregnancy
Interventions
Intravaneous iron(low molecular weight iron dextran)
Drug
Lead sponsor
Auerbach Hematology Oncology Associates P C
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 27, 2018 · Synced Sep 28, 2026, 3:15 PM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 2 Interventional Results available

T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies

NCT00683046
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Lymphoid Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma, Hodgkin's Disease, Chronic Lymphocytic Leukemia, Myeloproliferative Disorder, Anemia, Aplastic, Myelodysplastic Syndromes
Interventions
Fludarabine, Melphalan, Stem cells, Campath
Drug
Lead sponsor
University of Chicago
Other
Eligibility
Up to 100 Years
Enrollment
204 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2014
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 21, 2016 · Synced Sep 28, 2026, 3:15 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Haploidentical PBMC Transplant for Severe Congenital Anemias

NCT00977691
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
PBSC Transplant, Alemtuzumab, Sirolimus, Cyclophosphamide, Low Dose Irradiation
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Sep 28, 2026, 3:15 PM EDT
Completed No phase listed Observational

Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease

NCT01369160
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Quality of Life measures
Behavioral
Lead sponsor
Emory University
Other
Eligibility
3 Years to 23 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 27, 2014 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 2 Interventional

Umbilical Cord Blood Transplantation in Treating Patients With High-Risk Hematologic Cancer

NCT00003335
View directory record Official record
Conditions
Graft Versus Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclosporine, melphalan, methylprednisolone, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 54 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2012
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 14, 2012 · Synced Sep 28, 2026, 3:15 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant

NCT00964821
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis
Biological · Other
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 120 Years
Enrollment
459 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
Duarte, California • Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 6, 2017 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1 Interventional

Cyclophosphamide and/or Mycophenolate Mofetil With or Without Tacrolimus in Treating Patients Who Are Undergoing a Donor Bone Marrow or Peripheral Stem Cell Transplant for Hematologic Cancer

NCT00255710
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
filgrastim, cyclophosphamide, fludarabine phosphate, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 16, 2010 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1Phase 2 Interventional

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

NCT03653247
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Plerixafor, Busulfan, BIVV003
Biological · Drug · Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 40 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Not applicable Interventional Results available

Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

NCT00658385
View directory record Official record
Conditions
Beta Thalassemia Major, Congenital Anemias
Interventions
GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
Genetic
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 24, 2016 · Synced Sep 28, 2026, 3:15 PM EDT
Recruiting Phase 2 Interventional

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

NCT03579875
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure, Dyskeratosis Congenita, Telomere Biology Disorders
Interventions
Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion, G-CSF, Cyclophosphamide (CY) (Plan 2), Rituximab, Busulfan, Alemtuzumab, Melphalan
Drug · Device
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 65 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032
View directory record Official record
Conditions
Thalassemia Major With Severe Transfusional Iron Overload
Interventions
Deferasirox and deferiprone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 5, 2019 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 2 Interventional Results available

Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease

NCT01565616
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Conditioning Regimen with Bone Marrow Transplant
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years to 40 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2017 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Sep 28, 2026, 3:15 PM EDT
Terminated Phase 1 Interventional

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

NCT04443907
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
OTQ923
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 40 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Chicago, Illinois • New York, New York • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Sep 28, 2026, 3:15 PM EDT
Recruiting Phase 2 Interventional

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

NCT02828592
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Fludarabine, Cyclophosphamide, Total Body Irradiation, Rabbit ATG
Drug · Radiation
Lead sponsor
Northside Hospital, Inc.
Other
Eligibility
1 Year to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced Sep 28, 2026, 3:15 PM EDT
Terminated Phase 2 Interventional

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

NCT00034528
View directory record Official record
Conditions
Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
Interventions
Busulfan, Fludarabine, FK506, Prednisone
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 30, 2013 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 1 Interventional

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

NCT00578266
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Cyclophosphamide,Campath IH and TBI
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
Up to 60 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 23, 2017 · Synced Sep 28, 2026, 3:15 PM EDT
Completed Phase 2 Interventional

Chemotherapy Plus Biological Therapy Followed By Peripheral Stem Cell Transplantation in Treating Patients With Hematologic Cancer

NCT00004145
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
University of Chicago
Other
Eligibility
18 Years to 60 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2011
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Sep 28, 2026, 3:15 PM EDT
Active, not recruiting Phase 3 Interventional

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

NCT05600426
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG), rabbit anti-thymocyte globulin (ATG), Methotrexate, Fludarabine, Cyclophosphamide, low-dose total body irradiation (TBI), Immunosuppressive Therapy (IST)
Drug · Procedure · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Years to 25 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 44 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Sep 28, 2026, 3:15 PM EDT
Recruiting Phase 1Phase 2 Interventional

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

NCT04774536
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
CRISPR_SCD001
Drug
Lead sponsor
Mark Walters, MD
Other
Eligibility
12 Years to 35 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
2
States / cities
Los Angeles, California • Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Sep 28, 2026, 3:15 PM EDT