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Conditions: Hydrops Fetalis, Fetal Growth Retardation
Interventions: Tandem MS test for inborn errors of metabolism; Procedure
Lead sponsor: Obstetrix Medical Group; Industry
Eligibility: 18 Years to 60 Years · Female only
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 11
States / cities: Phoenix, Arizona • Laguna Hills, California • Long Beach, California + 6 more

Conditions: Transfusion Dependent Beta-thalassemia
Interventions: ST-400 Investigational product; Genetic
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 6
States / cities: Los Angeles, California • Oakland, California • Atlanta, Georgia + 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: 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: 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, 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: 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: 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: 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

Conditions: Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions: CliniMACS CD34 Reagent System; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: Up to 65 Years
U.S. locations: 1
States / cities: Durham, North Carolina

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: 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: 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: 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

Conditions: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions: Hematopoietic stem cell transplantation; Procedure
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: Up to 21 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia, Fetal Hydrops, Alpha; Thalassemia, Thalassemia Major, Thalassemia Alpha, A-Thalassemia
Interventions: in utero hematopoietic stem cell transplantation; Biological
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Weeks to 26 Weeks
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 1
States / cities: San Francisco, California

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: 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: Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
Interventions: Sangstat, Cyclophosphamide, Busulfan, Mycophenolate Mofetil, Cyclosporine, Cord Blood Transplantation; Drug · Procedure
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 3 Years to 14 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2006
U.S. locations: 14
States / cities: Oakland, California • Washington D.C., District of Columbia • Jacksonville, Florida + 10 more

Conditions: Sickle Cell Anemia, Beta Thalassemia
Interventions: TBI, Hydroxyurea, briquilimab, Filgrastim (G-CSF), Sirolimus, Alemtuzumab, Plerixafor; Radiation · Drug · Biological
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 4 Years to 100 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Congenital Hemolytic Anemia, Sickle Cell Disease
Interventions: Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus; Procedure · Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 80 Years
Enrollment: 130 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2026
U.S. locations: 1
States / cities: Bethesda, Maryland

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: 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: Iron Overload, Beta-Thalassemia
Interventions: SP-420; Drug
Lead sponsor: Sideris Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 3
States / cities: Boston, Massachusetts • New York, New York • Philadelphia, Pennsylvania

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Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

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

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

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

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

High-Tc Susceptometer to Monitor Transfusional Iron Overload

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

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Study of the Role of Genetic Modifiers in 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

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

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

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

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

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

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

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

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

Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia