Independent directory Public ClinicalTrials.gov records United States
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Showing 1–24 of 68 matching trials from the live ClinicalTrials.gov search.
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
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
Completed Phase 1 Interventional Accepts healthy volunteers

Safety, Tolerability and Pharmacokinetics of FTX-6058

NCT04586985
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