- 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
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Diamond Blackfan Anemia
- Interventions
- Sotatercept, Sotatercept with prednisone boost
- Drug
- Lead sponsor
- Northwell Health
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2021
- U.S. locations
- 1
- States / cities
- Manhasset, New York
- 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, 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
- Anemia, Leukemia, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
- Interventions
- Lenalidomide
- Drug
- Lead sponsor
- Jason Robert Gotlib
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Thalassemia
- Interventions
- Not listed
- Lead sponsor
- Carelon Research
- Other
- Eligibility
- 12 Years to 90 Years
- Enrollment
- 252 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 7
- States / cities
- Oakland, California • Atlanta, Georgia • Chicago, Illinois + 4 more
- Conditions
- Transfusion-dependent Anemia
- Interventions
- Deferasirox granule formulation, Deferasirox DT formulation
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 224 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 13
- States / cities
- Oakland, California • Atlanta, Georgia • Chicago, Illinois + 5 more
- Conditions
- Erythrocyte Transfusion, Beta-Thalassemia
- Interventions
- Luspatercept, Placebo
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 336 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
- Conditions
- RPS19 Deficient Diamond-Blackfan Anemia
- Interventions
- APR-2020
- Biological
- Lead sponsor
- Apriligen, Inc.
- Industry
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 2
- States / cities
- Stanford, California • Boston, Massachusetts
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Interventions
- thalidomide
- Drug
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Wilmington, North Carolina
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
- Interventions
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
- Drug · Radiation · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 50 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- 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
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Interventions
- Alemtuzumab, Total Body Irradiation, Cell Infusion, Thymoglobulin, Fludarabine, Busulfan, Cyclophosphamide, Sirolimus, Tacrolimus, Mycophenolate Mofetil, Plerixafor (mozobil)
- Drug · Radiation · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2032
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Diamond Blackfan Anemia, Pure Red Cell Aplasia
- Interventions
- Trifluoperazine
- Drug
- Lead sponsor
- Adrianna Vlachos, MD
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Manhasset, New York
- Conditions
- 5q Minus Syndrome, Myelodysplastic Syndrome
- Interventions
- Leucovorin
- Drug
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- Not listed
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 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
- Transfusion Dependent Thalassemia
- Interventions
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Device
- Lead sponsor
- Terumo BCTbio
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 4
- States / cities
- Oakland, California • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Interventions
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
- Biological · Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 5 Years to 40 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Myelodysplastic Syndrome
- Interventions
- Oral Azacitidine, Placebo, Best Supportiv Care (BSC)
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 216 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 40
- States / cities
- Berkeley, California • Beverly Hills, California • Duarte, California + 31 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
- Conditions
- Thalassemia
- Interventions
- AG-348
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2030
- U.S. locations
- 2
- States / cities
- Oakland, California • Boston, Massachusetts
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
- Interventions
- Dibotatug (DR-01)
- Drug
- Lead sponsor
- Dren Bio
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 10
- States / cities
- Duarte, California • Palo Alto, California • Miami, Florida + 7 more