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Conditions: Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia, Osteoporosis, Iron Overload, Hypertension, Pulmonary
Interventions: Deferoxamine, Deferiprone, Arginine, Sildenafil, Decitabine; Drug
Lead sponsor: Carelon Research; Other
Eligibility: 1 Year to 75 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2006
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • New York, New York + 1 more

Conditions: Beta-Thalassemia
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania

Conditions: Beta-Thalassemia
Interventions: No Intervention; Other
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Not listed
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2043
U.S. locations: 6
States / cities: Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more

Conditions: Beta Thalassemia
Interventions: 5-Azacytidine; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: Not listed
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

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

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions: data collection; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 60 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions: anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation; Drug · Procedure
Lead sponsor: Fairview University Medical Center; Other
Eligibility: 0 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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

Conditions: β-thalassemia Major
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 4
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more

Conditions: Beta-thalassemia, Iron Overload
Interventions: Deferasirox; Drug
Lead sponsor: Novartis; Industry
Eligibility: 10 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 3
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois

Conditions: Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Interventions: Not listed
Lead sponsor: Georgetown University; Other
Eligibility: 18 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2037
U.S. locations: 1
States / cities: San Francisco, California

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: MRI Scans
Interventions: Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI); Radiation
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 2 Years and older
Enrollment: 55 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2025
U.S. locations: 2
States / cities: New York, New York

Conditions: Transfusional Iron Overload, Beta-thalassemia
Interventions: FBS0701; Drug
Lead sponsor: FerroKin BioSciences, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2009
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Myelodysplastic Syndromes (MDS), Beta-thalassemia, Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
Interventions: Luspatercept; Drug
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 665 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2028
U.S. locations: 12
States / cities: Los Angeles, California • Oakland, California • Stanford, California + 9 more

Conditions: Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions: CD34 Stem Cell Selection Therapy; Biological
Lead sponsor: Diane George; Other
Eligibility: Up to 40 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Thalassemia
Interventions: Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Up to 64 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2016
U.S. locations: 1
States / cities: Houston, Texas

Conditions: β-thalassemia, Ineffective Erythropoiesis
Interventions: PTG-300; Drug
Lead sponsor: Protagonist Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 2
States / cities: Oakland, California • Boston, Massachusetts

Conditions: Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
Interventions: PROs assessment using sickle cell disease mobile app (SCD-app); Behavioral
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago; Other
Eligibility: 12 Years to 25 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Chicago, Illinois

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: Beta-Thalassemia
Interventions: ALS20; Biological
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 18 Years to 40 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2039
U.S. locations: 9
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 5 more

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Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

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

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

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

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

International Registry of Patients With Alpha Thalassemia

Study of the Role of Genetic Modifiers in Hemoglobinopathies

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Safety Study for Beta Thalassemia Subjects on PTG-300

Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

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

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

A Long-term Follow-up Study in Participants Who Received CTX001