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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Healthy Volunteers, Sickle Cell Disease
Interventions
FT-4202/Placebo
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
18
States / cities
Little Rock, Arkansas • Long Beach, California • Oakland, California + 12 more
Conditions
Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome With Excess Blasts, Myelodysplastic/Myeloproliferative Neoplasm, Myeloproliferative Neoplasm
Interventions
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Decitabine and Cedazuridine, Venetoclax
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
33
States / cities
Phoenix, Arizona • Costa Mesa, California • Irvine, California + 25 more
Conditions
Diabetes, Anemia
Interventions
TAP20-C, SAFE-T-FILL
Device
Lead sponsor
Seventh Sense Biosystems
Industry
Eligibility
18 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Cambridge, Massachusetts
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions
TER-1754, Placebo
Drug
Lead sponsor
Terremoto Biosciences Inc.
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Indianapolis, Indiana
Terminated No phase listed Observational

aHUS Observational Long Term Follow-Up

NCT01522170
Conditions
Atypical Hemolytic Uremic Syndrome
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
16
States / cities
Atlanta, Georgia • Dunwoody, Georgia • Fort Wayne, Indiana + 12 more
Conditions
Acute Leukemia, Myelodysplasia
Interventions
Unmanipulated Bone Marrow Graft with Tacrolimus/Methotrexate, Mobilized CD34-selected Peripheral Blood Stem Cell graft, Unmanipulated Bone Marrow Graft with Cyclophosphamide, Cyclophosphamide, Tacrolimus, Methotrexate
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Year to 65 Years
Enrollment
346 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
28
States / cities
Duarte, California • Stanford, California • Gainesville, Florida + 22 more
Conditions
Sickle Cell Disease
Interventions
Quality Improvement
Behavioral
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
715 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
Conditions
Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
RTI International
Other
Eligibility
1 Day to 31 Days
Enrollment
30,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Conditions
Anemia, Iron-Deficiency, Kidney Failure, Chronic
Interventions
Sodium Ferric Gluconate complex in sucrose, Ferrous sulfate tablets
Drug
Lead sponsor
Watson Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
23
States / cities
Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 20 more
Conditions
Sickle Cell Disease
Interventions
Osivelotor
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
24
States / cities
New Haven, Connecticut • Plantation, Florida • West Palm Beach, Florida + 11 more
Conditions
Sickle Cell Disease, Sickle Cell Anemia, SCD
Interventions
Fludarabine, Hematopoietic Stem Cell Transplant (HSCT)
Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
Interventions
Dibotatug (DR-01)
Drug
Lead sponsor
Dren Bio
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
9
States / cities
Duarte, California • Palo Alto, California • Atlanta, Georgia + 6 more
Conditions
Sickle Cell Disease
Interventions
Crovalimab, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years to 55 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Atlanta, Georgia • New York, New York
Conditions
Sickle Cell Disease
Interventions
massage, relaxation
Procedure · Behavioral
Lead sponsor
National Center for Complementary and Integrative Health (NCCIH)
NIH
Eligibility
15 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
2
States / cities
Los Angeles, California • Tampa, Florida
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat tablets
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
Cerritos, California • Los Angeles, California • Oakland, California + 9 more
Conditions
Shiga Toxin-Producing Escherichia Coli (E. Coli) Infection, Hemolytic-Uremic Syndrome
Interventions
Infusion of 200% maintenance fluids as balanced crystalloid IV solution, Oral fluids; infusion of up to 110% maintenance fluids as balanced crystalloid IV solution
Other
Lead sponsor
University of Calgary
Other
Eligibility
9 Months to 21 Years
Enrollment
1,040 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
22
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 19 more
Conditions
Leukemia, Myelodysplastic Syndromes
Interventions
paricalcitol
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
25 Years to 100 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts, Untreated Adult Acute Myeloid Leukemia, Myeloproliferative Neoplasm With 10% Blasts or Higher
Interventions
filgrastim, clofarabine, cytarabine
Biological · Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 64 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
3
States / cities
Duarte, California • Stanford, California • Seattle, Washington
Conditions
Healthy Volunteers Sickle Cell Disease, Thalassemia
Interventions
Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 55 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a), Childhood Acute Monocytic Leukemia (M5b), Childhood Acute Myeloblastic Leukemia With Maturation (M2), Childhood Acute Myeloblastic Leukemia Without Maturation (M1), Childhood Acute Myelomonocytic Leukemia (M4), Childhood Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Fanconi Anemia, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Ringed Sideroblasts, Secondary Myelodysplastic Syndromes, Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions
laboratory biomarker analysis
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Monrovia, California
Conditions
Malnutrition, Anemia
Interventions
MNP
Dietary Supplement
Lead sponsor
Columbia University
Other
Eligibility
6 Months to 54 Months
Enrollment
526 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
New York, New York
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Blood Collection for Research Related to Certain Diseases Involving Blood Vessels

NCT00047996
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2002
U.S. locations
2
States / cities
Bethesda, Maryland • Fairfax, Virginia
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Chronic Lymphocytic Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Myelodysplastic Syndromes, Stage III Chronic Lymphocytic Leukemia, Stage IV Chronic Lymphocytic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
R(+)XK469, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Houston, Texas
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
Interventions
CD34+ enriched, T Cell Depleted donor stem cell product
Biological
Lead sponsor
Christopher Dvorak
Other
Eligibility
2 Months to 30 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
San Francisco, California