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Conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions: Placebo Matching Mitapivat, Mitapivat; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 194 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 6
States / cities: La Jolla, California • Palo Alto, California • Boston, Massachusetts + 3 more

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

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: Thalassemia
Interventions: Luspatercept, Placebo, Best Supportive Care (BSC); Drug · Other
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 145 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 4
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois

Conditions: Beta-Thalassemia
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania

Conditions: Transfusion-dependent Beta-Thalassemia
Interventions: Safety and efficacy assessments; Other
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 66 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2035
U.S. locations: 6
States / cities: Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more

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: 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 Intermedia, Sickle Cell Disease
Interventions: Benserazide Only Product; Drug
Lead sponsor: Phoenicia BioScience; Other
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more

Conditions: Sickle Cell Disease, Thalassemia
Interventions: Etavopivat tablets; Drug
Lead sponsor: Forma Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 14
States / cities: Cerritos, California • Los Angeles, California • Oakland, California + 9 more

Conditions: Non-transfusion Dependent Thalassemia
Interventions: deferasirox, placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 10 Years and older
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2012
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • New York, New York

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

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: 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: 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: 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: Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions: Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen; Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2024
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Beta-Thalassemia
Interventions: VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo; Drug
Lead sponsor: Vifor (International) Inc.; Industry
Eligibility: 18 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Whittier, California

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

Conditions: Beta-Thalassemia
Interventions: LJPC-401; Drug
Lead sponsor: La Jolla Pharmaceutical Company; Industry
Eligibility: 14 Years and older
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Oakland, California • New York, New York

Conditions: Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions: REGN7999, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 6
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 2 more

Conditions: β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
Interventions: PTG-300; Drug
Lead sponsor: Protagonist Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Search by objective public record fields.

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

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 to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

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

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

The BENeFiTS Trial in Beta Thalassemia Intermedia

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

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

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

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

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

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

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

Pilot Study for Patients With Poor Response to Deferasirox

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia