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ClinicalTrials.gov public records Last synced Jul 23, 2026, 12:04 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Conditions
Overactive Bladder
Interventions
onaBoNT-A, Oxybutynin ER
Drug
Lead sponsor
Christopher Patrick Smith
Other
Eligibility
18 Years to 80 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Houston, Texas
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Analysis of Response of Subjects With Atopic Dermatitis or Psoriasis to Oral Vitamin D3

NCT00789880
Conditions
Atopic Dermatitis, Psoriasis
Interventions
Vitamin D3, Placebo
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 70 Years
Enrollment
82 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
3
States / cities
San Diego, California • Denver, Colorado • Portland, Oregon
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
VX-407, LNG/EE, NGM/EE, NET/EE, DRSP/EE
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 50 Years · Female only
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Overland Park, Kansas
Recruiting Not applicable Interventional

Efficacy of 3D-Printed Mouth Splints

NCT06801535
Conditions
Microstomia
Interventions
Splint
Device
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
2 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Conditions
Cystic Fibrosis
Interventions
Cysteamine, Placebo Oral Capsule
Drug
Lead sponsor
NovaBiotics Ltd.
Industry
Eligibility
18 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
8
States / cities
Tucson, Arizona • San Francisco, California • Gainesville, Florida + 5 more
Conditions
Prader-Willi Syndrome
Interventions
RAD011, Placebo
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
8
States / cities
Los Angeles, California • San Diego, California • Iowa City, Iowa + 5 more
Conditions
Cerebral Cavernous Malformations
Interventions
Propranolol, DNA and RNA Analysis
Drug · Genetic
Lead sponsor
St. Joseph's Hospital and Medical Center, Phoenix
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Phoenix, Arizona
Conditions
Adenocarcinoma, Adenocystic Carcinoma, Anal Cancer, Appendix Cancer, Brain Tumor, Glioblastoma, Astrocytoma, Bile Duct Cancer, Cholangiocarcinoma, Bladder Cancer, Bone Cancer, Synovial Sarcoma, Chondrosarcoma, Liposarcoma, Sarcoma, Kaposi, Sarcoma,Soft Tissue, Sarcoma, Osteosarcoma, CNS Cancer, Brain Stem Neoplasms, Breast Cancer, Cervical Cancer, Colorectal Cancer, Rectal Cancer, Colon Cancer, Esophageal Cancer, Esophagus Cancer, Cancer of Colon, Pancreatic Cancer, Cancer of Pancreas, Testis Cancer, Testicular Cancer, Ureter Cancer, Renal Cell Carcinoma, Kidney Cancer, Gestational Trophoblastic Tumor, Head and Neck Neoplasms, Parotid Tumor, Larynx Cancer, Tongue Cancer, Pharynx Cancer, Salivary Gland Cancer, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Multiple Myeloma, Non Hodgkin Lymphoma, Carcinoid Tumor, Lung Cancer, Neuroendocrine Tumors, Mesothelioma, Thyroid Cancer, Parathyroid Neoplasms, Adrenal Cancer, Small Bowel Cancer, Stomach Cancer, Liver Cancer, Hepatic Cancer, Melanoma, Skin Cancer, Unknown Primary Tumors, Uterine Cancer, Fallopian Tube Cancer, Ovarian Cancer, Prostate Cancer, Vaginal Cancer, Penile Cancer, Vulvar Cancer, Waldenstrom Macroglobulinemia, Cancer, Advanced, Thymus Cancer, Nasopharyngeal Carcinoma, Multiple Endocrine Neoplasia, Pheochromocytoma, Small Cell Carcinoma, Pulmonary Carcinoma
Interventions
Biomarker Testing (L), Systemic Treatment (T), Patient Reported Outcomes (P)
Diagnostic Test · Drug · Other
Lead sponsor
Taproot Health
Industry
Eligibility
18 Years and older
Enrollment
167 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Idaho Falls, Idaho • Laredo, Texas
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Erythroplakia, Fanconi Anemia, Lichen Planus, Oral Cavity and Lip Precancerous Condition, Oral Cavity Leukoplakia, Oral Neoplasm, Premalignant Lesion, Tobacco Use Disorder
Interventions
Cytology Specimen Collection Procedure, Diagnostic Microscopy, Diagnostic Procedure, Fluorescence Imaging, Fluorescence Spectroscopy
Other · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2027
U.S. locations
1
States / cities
Houston, Texas
Conditions
Anemia, Sickle Cell, Pneumonia
Interventions
Arginine hydrochloride
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
5 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
1
States / cities
Oakland, California
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Fargo, North Dakota
Conditions
Cytomegalovirus Infections
Interventions
Valganciclovir, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Up to 30 Days
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
21
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 18 more
Conditions
Primary Ciliary Dyskinesia
Interventions
VX-371, Hypertonic Saline, Placebo (0.17% saline), VX-371 + HS, Ivacaftor
Drug
Lead sponsor
Parion Sciences
Industry
Eligibility
12 Years and older
Enrollment
123 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
21
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 18 more
Conditions
Exocrine Pancreatic Insufficiency, Chronic Pancreatitis, Cystic Fibrosis
Interventions
Pancrelipase
Drug
Lead sponsor
Digestive Care, Inc.
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Completed No phase listed Observational

Study of Smith-Lemli-Opitz Syndrome

NCT00001721
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholesterol Suspension, CRH, Cholesterol
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hereditary Angioedema (HAE), Angioedema, Bradykinin-mediated Angioedema, C1 Inhibitor Deficiency
Interventions
Deucrictibant XR tablet, Placebo comparator to XR tablet, Deucrictibant 20 mg capsule, Placebo comparator to 20 mg capsule
Drug
Lead sponsor
Institute for Asthma and Allergy
Network
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Wheaton, Maryland
Conditions
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions
RAY121
Drug
Lead sponsor
Chugai Pharmaceutical
Industry
Eligibility
18 Years to 85 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Orange, California • Baltimore, Maryland • Lake Success, New York + 8 more
Completed Not applicable Interventional

Feeding Progression in Preterm Infants

NCT02027688
Conditions
Infant, Premature, Diseases, Feeding Behavior, Feeding Patterns
Interventions
q6 hour oral feeding schedule, q3 hour oral feeding schedule
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Up to 120 Days
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Conditions
Prader-Willi Syndrome
Interventions
ARD-101
Drug
Lead sponsor
Aardvark Therapeutics, Inc.
Industry
Eligibility
17 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Palo Alto, California • Aurora, Colorado
Conditions
Fabry Disease
Interventions
Lucerastat
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
14
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 10 more
Conditions
Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, de Novo Myelodysplastic Syndromes, Disseminated Neuroblastoma, Juvenile Myelomonocytic Leukemia, Mucositis, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Childhood Rhabdomyosarcoma, Previously Treated Myelodysplastic Syndromes, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Recurrent/Refractory Childhood Hodgkin Lymphoma, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
supersaturated calcium phosphate rinse, placebo, questionnaire administration, quality-of-life assessment
Drug · Other · Procedure
Lead sponsor
Children's Oncology Group
Network
Eligibility
4 Years to 21 Years
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
31
States / cities
Arcadia, California • Duarte, California • Oakland, California + 26 more
Completed Early Phase 1 Interventional Results available

A Dose-Finding Study of AG-348 in Sickle Cell Disease

NCT04000165
Conditions
Sickle Cell Disease
Interventions
AG-348
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 70 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland