- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Interventions
- Exa-cel
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 9
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 4 more
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.
All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 68
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Sickle Cell Disease
- Interventions
- Zileuton
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Renal Impairment
- Interventions
- GBT021601
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 4
- States / cities
- Lake Forest, California • Tustin, California • Miami, Florida + 1 more
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
- Interventions
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 3 Months to 39 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Sickling Disorder Due to Hemoglobin S
- Interventions
- 131I-apamistmab, Sirolimus, Campath, Total Body Irradiation, Exchange Transfusion, Planar gamma imaging
- Drug · Radiation · Procedure
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease
- Interventions
- nula-cel Drug Product
- Genetic
- Lead sponsor
- Kamau Therapeutics
- Industry
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Palo Alto, California • St Louis, Missouri + 2 more
- Conditions
- Healthy, Sickle Cell Anemia
- Interventions
- Placebo, PF-07209326
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 16 Years to 70 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2023
- U.S. locations
- 16
- States / cities
- New Haven, Connecticut • Washington D.C., District of Columbia • Fort Myers, Florida + 6 more
- Conditions
- Sickle Cell Disease
- Interventions
- BEAM-101
- Biological
- Lead sponsor
- Beam Therapeutics Inc.
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Jacksonville, Florida + 14 more
- Conditions
- Sickle Cell Disease
- Interventions
- Plerixafor, Busulfan, BIVV003
- Biological · Drug · Genetic
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years to 40 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 5
- States / cities
- Oakland, California • Sacramento, California • Atlanta, Georgia + 2 more
- Conditions
- Healthy Adult Subjects, Sickle Cell Disease
- Interventions
- FTX-6058/placebo oral capsule(s), FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup
- Drug
- Lead sponsor
- Fulcrum Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 109 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2022
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Overland Park, Kansas
- Conditions
- Sickle Cell Disease
- Interventions
- Placebo, Zoledronic Acid
- Drug
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 18 Years to 70 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- Sickle Cell Disease
- Interventions
- Femoral Nerve Block
- Procedure
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease
- Interventions
- Intravenous citrulline
- Drug
- Lead sponsor
- University of Mississippi Medical Center
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Jackson, Mississippi
- Conditions
- Sickle Cell Anemia
- Interventions
- Measurement of carbon monoxide level in exhaled breath
- Other
- Lead sponsor
- Ashutosh Lal
- Other
- Eligibility
- 5 Years to 14 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Sickle Cell Disease, Hemolytic Anemia
- Interventions
- Mitapivat
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hemoglobin SC Disease, Sickle Cell Anemia
- Interventions
- Recombinant-methionyl human stem cell factor
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sickle Cell Disease, Hemoglobinopathies
- Interventions
- EDIT-301
- Genetic
- Lead sponsor
- Editas Medicine, Inc.
- Industry
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 21
- States / cities
- Oakland, California • Aurora, Colorado • New Haven, Connecticut + 15 more
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Interventions
- Pociredir oral capsule(s)
- Drug
- Lead sponsor
- Fulcrum Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 14
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Atlanta, Georgia + 11 more
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Sickle Cell Hemoglobin C Disease, Pain Crisis
- Interventions
- Buprenorphine Buccal Film [Belbuca]
- Drug
- Lead sponsor
- Natasha Archer
- Other
- Eligibility
- 12 Years to 22 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2027 – 2032
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Healthy Volunteers, Anemia, Sickle Cell
- Interventions
- AG-946, AG-946 Matched Placebo
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 122 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2023
- U.S. locations
- 8
- States / cities
- La Jolla, California • Boston, Massachusetts • New York, New York + 4 more
- Conditions
- Sickle Cell Disease
- Interventions
- Allogeneic Non-Myeloablative Stem Cell Transplantation, Alemtuzumab, Sirolimus
- Procedure · Drug
- Lead sponsor
- University of Illinois Chicago
- Other
- Eligibility
- 16 Years to 60 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2029
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Sickle Cell Disease
- Interventions
- CSL889
- Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 8
- States / cities
- Chicago, Illinois • Baltimore, Maryland • Minneapolis, Minnesota + 5 more
- Conditions
- Sickle Cell Disease
- Interventions
- GBT440
- Drug
- Lead sponsor
- Global Blood Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- San Antonio, Texas