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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 55 Years
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Beta Thalassemia, Hemoglobinopathy
Interventions: Hydroxyurea; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

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
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: 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: Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions: REGN7999, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 95 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
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: 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: 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, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
Interventions: EDIT-301; Genetic
Lead sponsor: Editas Medicine, Inc.; Industry
Eligibility: 18 Years to 35 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 7
States / cities: Oakland, California • Minneapolis, Minnesota • New York, New York + 3 more

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: 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: 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 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: 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

Conditions: Beta-Thalassemia
Interventions: Deferitrin (GT56-252), desferoxamine (DFO); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2005
U.S. locations: 1
States / cities: New York, New York

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: Sickle Cell Anemia
Interventions: Ambrisentan, Placebo; Drug
Lead sponsor: Augusta University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Augusta, Georgia

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: 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: Transfusional Iron Overload, Beta-Thalassemia
Interventions: SPD602; Drug
Lead sponsor: Shire; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 2
States / cities: Boston, Massachusetts • Philadelphia, Pennsylvania

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: 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: Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
Interventions: Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi); Drug
Lead sponsor: New York Medical College; Other
Eligibility: Up to 70 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2018
U.S. locations: 1
States / cities: Valhalla, New York

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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Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

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

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

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

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

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

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

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

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

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

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

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

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

The Role of Endothelin-1 in 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

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

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

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

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

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