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Showing 1–24 of 67 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Phase 1 Sickle Cell Clear all
Completed Phase 1 Interventional Results available

Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease

NCT02114203
View directory record Official record
Conditions
Phase 1 Sickle Cell
Interventions
PDE9i, placebo for PDE9i
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
12
States / cities
Chicago, Illinois • Boston, Massachusetts • Brooklyn, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2017 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional

A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease

NCT01597401
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Aes-103, Placebo
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 4, 2021 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease

NCT04285827
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 5, 2023 · Synced Jul 24, 2026, 7:04 PM EDT
Withdrawn Phase 1 Interventional

US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease

NCT04594031
View directory record Official record
Conditions
Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
Interventions
ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Biological
Lead sponsor
ExCellThera inc.
Industry
Eligibility
5 Years to 30 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Palo Alto, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 4, 2022 · Synced Jul 24, 2026, 7:04 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

NCT04853576
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 30, 2025 · Synced Jul 24, 2026, 7:04 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 24, 2026, 7:04 PM EDT
Terminated Phase 1 Interventional

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

NCT04443907
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
OTQ923
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 40 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Chicago, Illinois • New York, New York • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

NCT05169580
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

NCT05904093
View directory record Official record
Conditions
Sickle Cell Disease, Hb-SS Disease, Hemoglobin S, Disease Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin Beta Thalassemia Disease
Interventions
Fostamatinib
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function

NCT06145282
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Briquilimab, Filgrastim, Abatacept, Sirolimus, Cyclophosphamide
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety, Tolerability and Pharmacokinetics of FTX-6058

NCT04586985
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 11, 2022 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects

NCT02567695
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
GBT440
Drug
Lead sponsor
Global Blood Therapeutics
Industry
Eligibility
18 Years to 60 Years
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Apr 11, 2017 · Synced Jul 24, 2026, 7:04 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

NCT04610866
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Terminated Phase 1 Interventional

A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease

NCT03132324
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
INCB059872
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
7
States / cities
Miami, Florida • Tamarac, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2019 · Synced Jul 24, 2026, 7:04 PM EDT
Terminated Phase 1 Interventional

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 45 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Jul 24, 2026, 7:04 PM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

NCT06481306
View directory record Official record
Conditions
Anemia, Sickle Cell, Healthy Volunteers
Interventions
BMS-986470, Placebo, Famotidine, Pantoprazole
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
224 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2029
U.S. locations
19
States / cities
Birmingham, Alabama • La Jolla, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional Results available

A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome

NCT02697240
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 24, 2019 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects

NCT02567682
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 11, 2017 · Synced Jul 24, 2026, 7:04 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Omega-3 Fatty Acids in Sickle Cell Disease

NCT02947100
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
SCD-Omegatex™
Drug
Lead sponsor
Robin E. Miller
Other
Eligibility
8 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Oct 18, 2020 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
lovo-cel
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional

Study of Decitabine and Tetrahydrouridine (THU) in Patients With Sickle Cell Disease

NCT01685515
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Oral Decitabine and Tetrahydrouridine, Placebo
Drug
Lead sponsor
Yogen Saunthararajah
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 10, 2018 · Synced Jul 24, 2026, 7:04 PM EDT
Completed Phase 1 Interventional

Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)

NCT01783691
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
NKTT120
Drug
Lead sponsor
NKT Therapeutics
Industry
Eligibility
18 Years to 60 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
6
States / cities
Oakland, California • Baltimore, Maryland • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2015 · Synced Jul 24, 2026, 7:04 PM EDT
Recruiting Phase 1Phase 2 Interventional

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996
View directory record Official record
Conditions
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
RIC regimen, GVHD prophylaxis regimen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2033
U.S. locations
4
States / cities
New Haven, Connecticut • Wilmington, Delaware • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 24, 2026, 7:04 PM EDT