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Conditions: Beta-Thalassemia
Interventions: ICL670, deferoxamine; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 595 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 7
States / cities: Los Angeles, California • Oakland, California • Stanford, California + 4 more

Conditions: Sickle Cell Disease, Sickle Cell Anemia
Interventions: vorinostat; Drug
Lead sponsor: Dana-Farber Cancer Institute; Other
Eligibility: 18 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 3
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: 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: 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: Thalassemia Major
Interventions: DST-0509, Jadenu, Exjade; Drug
Lead sponsor: DisperSol Technologies, LLC; Industry
Eligibility: 8 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
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 – 2026
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
Interventions: IMR-687, Placebo Oral Capsule; Drug
Lead sponsor: Cardurion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 66 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Overland Park, Kansas

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: 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: 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: Thalassemia Major
Interventions: Not listed
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago; Other
Eligibility: 0 Years to 30 Years
Enrollment: 176 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
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: 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: 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: 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: 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: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

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: 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: Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, AML, TP53, Intrachromosomal Amplification of Chromosome 21, Cytogenetic Abnormality, CNS Leukemia, Minimal Residual Disease, Myelodysplasia, Juvenile Myelomonocytic Leukemia, Somatic Mutation, PTPN11 Gene Mutation, N-RAS Gene Amplification, Neurofibromatosis 1, NF1 Mutation, CBL Gene Mutation, Monosomy 7, Chromosome Abnormality, Fetal Hemoglobin, Lymphoblastic Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult
Interventions: Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam, Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells, Thymoglobulin, Cyclophosphamide; Drug · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 60 Years
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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: 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: 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: 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

Search by objective public record fields.

Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

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

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

The Role of Endothelin-1 in Sickle Cell Disease

Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation

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

A Study of IMR-687 in Healthy Adult Volunteers

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

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

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

Long Term Outcomes in β Thalassemia Major

High-Tc Susceptometer to Monitor Transfusional Iron Overload

Pilot Study for Patients With Poor Response to Deferasirox

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

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

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

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

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

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

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

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

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

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