Search | Clinical Trials Finder

Conditions: Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
Interventions: GWAS; Genetic
Lead sponsor: Cyprus Institute of Neurology and Genetics; Other
Eligibility: 2 Years and older
Enrollment: 30,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions: Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab; Drug · Biological
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 2 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 6
States / cities: Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more

Conditions: Sickle Cell Disease, Beta Thalassemia
Interventions: Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant; Drug · Procedure
Lead sponsor: Columbia University; Other
Eligibility: 1 Month to 30 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2025
U.S. locations: 1
States / cities: New York, New York

Conditions: Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 300 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2029
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions: Not listed
Lead sponsor: xCures; Industry
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Los Altos, California

Conditions: Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
Interventions: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452; Biological · Device
Lead sponsor: Daniel Bauer; Other
Eligibility: 13 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions: HU-Go app; Other
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago; Other
Eligibility: 12 Years to 25 Years
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Chicago, Illinois

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

Conditions: Sickle Cell Disease, Beta-thalassemia
Interventions: Total Body Irradiation, Alemtuzumab, Abatacept, Research blood draw; Radiation · Drug · Other
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 4 Years to 65 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2035
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Myeloid Chimerism
Interventions: CliniMACS CD34 Reagent; Device
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 80 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2037
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Thalassemia Major
Interventions: CliniMACS; Device
Lead sponsor: Timothy Olson; Other
Eligibility: 2 Years to 25 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Sickle Cell Disease
Interventions: Pociredir; Drug
Lead sponsor: Fulcrum Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 8
States / cities: Little Rock, Arkansas • Los Angeles, California • Baton Rouge, Louisiana + 5 more

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

Conditions: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 3
States / cities: New York, New York • Charlotte, North Carolina • Nashville, Tennessee

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

Conditions: Sickle Cell Disease
Interventions: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a; Biological
Lead sponsor: David Williams; Other
Eligibility: 13 Years to 40 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 9
States / cities: Los Angeles, California • Oakland, California • Sacramento, California + 4 more

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

Conditions: Hemoglobin SC Disease, Sickle Cell Anemia
Interventions: Recombinant-methionyl human stem cell factor; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: Not listed
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Pociredir; Drug
Lead sponsor: Fulcrum Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 6
States / cities: Miami, Florida • Orlando, Florida • Riverdale, Georgia + 3 more

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

Conditions: Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
Interventions: Specimen sample; Diagnostic Test
Lead sponsor: Sanguine Biosciences; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 20,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Waltham, Massachusetts

Conditions: Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 16 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
Interventions: MED-Go App; Behavioral
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago; Other
Eligibility: 12 Years to 21 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions: Enriched Hematopoetic Stem Cell Infusion; Biological
Lead sponsor: Talaris Therapeutics Inc.; Industry
Eligibility: Up to 45 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina

Search by objective public record fields.

Study of the Role of Genetic Modifiers in Hemoglobinopathies

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

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

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

Open-Label Extension Study to Pioneer Study 6058-SCD-101

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

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

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

A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

High-Tc Susceptometer to Monitor Transfusional Iron Overload

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies