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Showing 1–24 of 2,339 matching trials from the live ClinicalTrials.gov search.
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Completed Early Phase 1 Interventional

PET/MRI Evaluation of Cardiac Amyloid

NCT03119558
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
18F-Florbetaben (Neuraceq®) PET/MRI
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jul 16, 2019 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Early Phase 1 Interventional

Pemetrexed Disodium in the Cerebrospinal Fluid of Patients With Leptomeningeal Metastases

NCT00424242
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Metastatic Cancer, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Secondary Myelofibrosis, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Pemetrexed
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 120 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 7, 2021 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

The Diet Gout Trial

NCT03569020
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Subsidy for food purchases and dietitian education
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
2
States / cities
Woodlawn, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 29, 2021 · Synced Jun 10, 2026, 9:33 PM EDT
Recruiting Phase 3 Interventional

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl-L-Leucine, Placebo
Drug · Other
Lead sponsor
IntraBio Inc
Industry
Eligibility
4 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Early Phase 1 Interventional

Methylprednisolone Treatment of Friedreich Ataxia

NCT02424435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Methylprednisolone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 24, 2021 · Synced Jun 10, 2026, 9:33 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

NCT00962260
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
U.S. locations
14
States / cities
La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 4 Interventional Results available

Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

NCT04026178
View directory record Official record
Conditions
Generalized Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Birmingham, Alabama • New Orleans, Louisiana • Ann Arbor, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2025 · Synced Jun 10, 2026, 9:33 PM EDT
Completed No phase listed Observational

The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization, Multiple Basal Cell Carcinomas and Other Retinoid Responsive Diseases

NCT00005660
View directory record Official record
Conditions
Basal Cell Carcinoma, Keratosis Palmaris et Plantaris, Psoriasis
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1977 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 12, 2021 · Synced Jun 10, 2026, 9:33 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

A Bifido Bacteria to Improve Lactose Digestion and Tolerance

NCT05668468
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Bifidobacterium adolescentis IVS-1 capsule, Placebo cellulose capsule
Dietary Supplement · Other
Lead sponsor
Purdue University
Other
Eligibility
18 Years to 65 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 28, 2022 · Synced Jun 10, 2026, 9:33 PM EDT
Recruiting No phase listed Observational

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 16 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2025 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced Jun 10, 2026, 9:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Clinical and Genetic Studies of Li-Fraumeni Syndrome

NCT01443468
View directory record Official record
Conditions
Li-Fraumeni Syndrome, Neoplasms, Tp53 Mutations
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 4 Interventional Results available

Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study

NCT01735955
View directory record Official record
Conditions
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
Interventions
Nilotinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Albany, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 2 Interventional

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

NCT00051935
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Jun 10, 2026, 9:33 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Comparing the Adaptation of Commercial Milk and A2 Milk in Lactose Maldigesters

NCT05669274
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Milk containing A1 and A2 beta-casein, Milk containing only A2 beta-casein
Other
Lead sponsor
Purdue University
Other
Eligibility
18 Years to 65 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 29, 2023 · Synced Jun 10, 2026, 9:33 PM EDT
Withdrawn Phase 2 Interventional

Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome

NCT00514982
View directory record Official record
Conditions
Hermanski-Pudlak Syndrome, Colitis, Cytokines, Lymphocytes, Drug Evaluation
Interventions
Mesalamine, Infliximab, Corticosteroids, 6-Mercaptopurine, Tacrolimus, Adalimumab
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 4 Interventional Results available

Effects of Hyperuricemia Reversal on Features of the Metabolic Syndrome

NCT01654276
View directory record Official record
Conditions
Gout
Interventions
Febuxostat
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
21 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 13, 2023 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049
View directory record Official record
Conditions
Mucopolysaccharidosis IIIB
Interventions
SBC-103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 20, 2018 · Synced Jun 10, 2026, 9:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jun 10, 2026, 9:33 PM EDT
Not yet recruiting No phase listed Observational

Clinical Genetics Branch Eligibility Screening Survey

NCT07005297
View directory record Official record
Conditions
Melanoma, Li-Fraumeni Syndrome, Pulmonary Blastoma, Chordoma, Congenital Bone Marrow Failure Syndromes, Costello Syndrome, Fanconi Anemia, CFC Syndrome (CFCS), Legius Syndrome, RASopathies
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jun 10, 2026, 9:33 PM EDT
Completed No phase listed Observational

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220
View directory record Official record
Conditions
Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Interventions
There are no interventions in this study.
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced Jun 10, 2026, 9:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Li-Fraumeni Syndrome/TP53 Biobank

NCT04367246
View directory record Official record
Conditions
Li-Fraumeni Syndrome, Li-Fraumeni-Like Syndrome
Interventions
No Intervention
Other
Lead sponsor
Abramson Cancer Center at Penn Medicine
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Phildelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 13, 2025 · Synced Jun 10, 2026, 9:33 PM EDT
Completed Phase 2 Interventional Results available

Stannsoporfin With Light Therapy for Newborn Babies With Jaundice

NCT01887327
View directory record Official record
Conditions
Jaundice, Neonatal, Hyperbilirubinemia, Neonatal
Interventions
Phototherapy, Stannsoporfin, Placebo
Procedure · Drug
Lead sponsor
InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
Industry
Eligibility
1 Hour to 72 Hours
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
18
States / cities
Colton, California • San Diego, California • San Francisco, California + 14 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2020 · Synced Jun 10, 2026, 9:33 PM EDT