- 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 Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–35
of 35
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Interventions
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Biological · Device
- Lead sponsor
- Daniel Bauer
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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