- 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
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- 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
- 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
- Beta-Thalassemia
- Interventions
- LJPC-401
- Drug
- Lead sponsor
- La Jolla Pharmaceutical Company
- Industry
- Eligibility
- 14 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • New York, New York
- 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
- 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
- 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
- Thalassemia
- Interventions
- Luspatercept, Placebo, Best Supportive Care (BSC)
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 145 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois
- 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