Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Jun 26, 2026, 6:43 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Conditions
Prader-Willi Syndrome, Obesity
Interventions
Group 1, Group 2
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 60 Years
Enrollment
392 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
4
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 1 more
Conditions
Hyperbilirubinemia, Jaundice
Interventions
Gene mutation sample
Procedure
Lead sponsor
Pediatrix
Other
Eligibility
Up to 6 Days
Enrollment
450 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2007
U.S. locations
1
States / cities
Greenville, South Carolina
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
2 Years to 99 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Metabolic Syndrome
Interventions
ELM Group, ELM Classes, ELM Individual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
18 Years to 72 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Gout
Interventions
Lesinurad, Placebo, Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
607 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
165
States / cities
Athens, Alabama • Birmingham, Alabama • Gulf Shores, Alabama + 139 more
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Food Effect Study of Febuxostat XR in Healthy Participants

NCT02374164
Conditions
Gout
Interventions
Febuxostat XR
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years to 55 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
1
States / cities
Austin, Texas
Not listed No phase listed Observational Accepts healthy volunteers

Insight Into Subcutaneous Adipose Tissue Disorders

NCT02838277
Conditions
Lipedema, Dercum's Disease, Familial Multiple Lipomatosis, Madelung's Disease
Interventions
Not listed
Lead sponsor
University of Arizona
Other
Eligibility
19 Years to 70 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
PBGM01
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 24 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
Conditions
Pompe Disease (Late-onset)
Interventions
AT2221, ATB200
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
18
States / cities
Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more
Conditions
Polycystic Ovary Syndrome
Interventions
Salsalate
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Fairfax, Virginia
Conditions
Hypophosphatasia (HPP)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
9
States / cities
Atlanta, Georgia • Indianapolis, Indiana • Boston, Massachusetts + 6 more
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Conditions
Propionic Acidemia
Interventions
mRNA-3927
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
15
States / cities
Los Angeles, California • Stanford, California • Miami, Florida + 11 more
Not listed Not applicable Interventional Accepts healthy volunteers

A Bifido Bacteria to Improve Lactose Digestion and Tolerance

NCT05668468
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
Conditions
Prader-Willi Syndrome, Obesity, Hyperphagia
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Gainesville, Florida
Available No phase listed Expanded access

Expanded Access Metreleptin Study

NCT02404896
Conditions
Familial Partial Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Stanford, California • New York, New York • Pittsburgh, Pennsylvania
Conditions
Digestive Health, Abdominal Discomfort, Gas and Bloating
Interventions
Bloat Gummies, Placebo
Dietary Supplement
Lead sponsor
Arrae
Industry
Eligibility
18 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
San Francisco, California
Conditions
Gout
Interventions
Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
1,790 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
192
States / cities
Binghamton, Alabama • Birmingham, Alabama • Huntsville, Alabama + 189 more
Conditions
Prader-Willi Syndrome
Interventions
DCCR, Placebo for DCCR
Drug
Lead sponsor
Soleno Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
20
States / cities
Orange, California • Palo Alto, California • San Diego, California + 17 more