- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
- Interventions
- Enriched Hematopoetic Stem Cell Infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- Up to 45 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
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
- 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
- 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 – 2027
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- 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
- 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
- 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
- Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
- Interventions
- Not listed
- Lead sponsor
- Georgetown University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Non-Transfusion Dependent Beta-Thalassemia (NTDT)
- Interventions
- REGN7999, Placebo
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- 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
- Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat Matched Placebo, Mitapivat
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 4
- States / cities
- Atlanta, Georgia • New York, New York • Philadelphia, Pennsylvania + 1 more