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Showing 1–24 of 75 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Sickle Cell Disease Clear all
Completed No phase listed Observational Accepts healthy volunteers

QST-Pupillometry in Sickle Cell Disease Patients

NCT02242058
View directory record Official record
Conditions
SCD With Severe Phenotype (HbSS, HbSβ0 Thalassemia, HbSOARab)
Interventions
Quantitative sensory testing
Other
Lead sponsor
Julia Finkel
Other
Eligibility
13 Years to 30 Years
Enrollment
96 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 20, 2020 · Synced Jun 10, 2026, 8:59 PM EDT
Withdrawn Phase 1 Interventional

US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease

NCT04594031
View directory record Official record
Conditions
Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
Interventions
ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Biological
Lead sponsor
ExCellThera inc.
Industry
Eligibility
5 Years to 30 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Palo Alto, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 4, 2022 · Synced Jun 10, 2026, 8:59 PM EDT
Not listed Not applicable Interventional

Study of an Intervention to Improve Problem List Accuracy and Use

NCT01105923
View directory record Official record
Conditions
Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer, Coronary Artery Disease, Congestive Heart Failure, Diabetes, Glaucoma, Hemophilia, Hypertension, Hyperthyroidism, Hypothyroidism, Myasthenia Gravis, Osteoporosis, Osteopenia, Renal Failure, Renal Insufficiency, Sickle Cell Disease, Stroke
Interventions
MAPLE
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 1, 2015 · Synced Jun 10, 2026, 8:59 PM EDT
Recruiting Phase 1Phase 2 Interventional

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996
View directory record Official record
Conditions
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
RIC regimen, GVHD prophylaxis regimen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2033
U.S. locations
4
States / cities
New Haven, Connecticut • Wilmington, Delaware • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jun 10, 2026, 8:59 PM EDT
Completed Phase 2 Interventional Results available

Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

NCT00004412
View directory record Official record
Conditions
Skin Ulcers, Sickle Cell Anemia
Interventions
Arginine Butyrate, Standard local care dressing
Drug · Other
Lead sponsor
Susan P. Perrine
Other
Eligibility
16 Years to 60 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2005
U.S. locations
4
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced Jun 10, 2026, 8:59 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 3, 2024 · Synced Jun 10, 2026, 8:59 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 25, 2019 · Synced Jun 10, 2026, 8:59 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 Jun 10, 2026, 8:59 PM 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 1, 2025 · Synced Jun 10, 2026, 8:59 PM EDT
Terminated Phase 2 Interventional Results available

Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT01000155
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
vorinostat
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
3
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 20, 2017 · Synced Jun 10, 2026, 8:59 PM EDT
Recruiting Phase 1 Interventional

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

NCT07015684
View directory record Official record
Conditions
Sickling Disorder Due to Hemoglobin S
Interventions
131I-apamistmab, Sirolimus, Campath, Total Body Irradiation, Exchange Transfusion, Planar gamma imaging
Drug · Radiation · Procedure
Lead sponsor
Columbia University
Other
Eligibility
12 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jun 10, 2026, 8:59 PM EDT
Completed No phase listed Observational

Analysis of T-Cell Immune Reconstitution Following Allogeneic Hematopoietic BMT for Severe SCD

NCT00228631
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
6 Months to 21 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 25, 2014 · Synced Jun 10, 2026, 8:59 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 Jun 10, 2026, 8:59 PM EDT
Terminated Phase 2 Interventional

Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide

NCT00041574
View directory record Official record
Conditions
Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant, Lung Transplant
Interventions
Inhaled Nitric Oxide
Drug
Lead sponsor
Mallinckrodt
Industry
Eligibility
5 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
3
States / cities
Denver, Colorado • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Sep 8, 2016 · Synced Jun 10, 2026, 8:59 PM EDT
Terminated Phase 1 Interventional

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 45 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Jun 10, 2026, 8:59 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 26, 2020 · Synced Jun 10, 2026, 8:59 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

NCT00040469
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
Campath -1H, Dilantin, Busulfan, Cyclophosphamide
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 64 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 17, 2020 · Synced Jun 10, 2026, 8:59 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 Jun 10, 2026, 8:59 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 Jun 10, 2026, 8:59 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

NCT00040417
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
FLUDARABINE, CAMPATH-IH, Total Body Irradiation, FK506, G-SCF (Granulocyte-colony stimulating factor)
Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 17, 2020 · Synced Jun 10, 2026, 8:59 PM EDT
Terminated Phase 1 Interventional Results available

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

NCT02231710
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 24, 2024 · Synced Jun 10, 2026, 8:59 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

High-Tc Susceptometer to Monitor Transfusional Iron Overload

NCT01241357
View directory record Official record
Conditions
Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes, Aplastic Anemia
Interventions
Hepatic biomagnetic susceptibility measurement
Device
Lead sponsor
Columbia University
Other
Eligibility
5 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 20, 2016 · Synced Jun 10, 2026, 8:59 PM EDT
Completed Phase 4 Interventional Results available

Pilot Study for Patients With Poor Response to Deferasirox

NCT00749515
View directory record Official record
Conditions
Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
Interventions
Deferoxamine, Deferasirox, HIDA
Drug · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 11, 2024 · Synced Jun 10, 2026, 8:59 PM EDT
Completed Phase 2 Interventional Results available

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

NCT00004143
View directory record Official record
Conditions
Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions
Campath, Chemo and/or TBI Allo SCT
Drug
Lead sponsor
David Rizzieri, MD
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
2
States / cities
Orlando, Florida • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 4, 2014 · Synced Jun 10, 2026, 8:59 PM EDT