Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 9, 2026, 3:34 AM EDT

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Showing 73–96 of 1,599 matching trials from the live ClinicalTrials.gov search.
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Hypophosphatasia
Interventions
Whole Genome Sequencing
Genetic
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
Not listed
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Kansas City, Missouri
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Conditions
Porphyria
Interventions
luteinizing hormone-releasing factor
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1987
U.S. locations
1
States / cities
Galveston, Texas
Conditions
Congenital Adrenal Hyperplasia
Interventions
Pre-Screening
Other
Lead sponsor
Adrenas Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Active, not recruiting No phase listed Observational

Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Biological
Lead sponsor
John Bernat
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2037
U.S. locations
1
States / cities
Iowa City, Iowa
Conditions
Sanfilippo Syndrome Type A
Interventions
GC1130A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
12 Months to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Conditions
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium
Drug
Lead sponsor
Astra USA
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Houston, Texas
Conditions
Pelizaeus-Merzbacher Disease, PMD
Interventions
HuCNS-SC transplant in the lead-in phase
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Conditions
Sanfilippo Syndrome
Interventions
Recombinant human heparan N-sulfatase [rhHNS]
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Torrance, California • Minneapolis, Minnesota • Chapel Hill, North Carolina
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
Interventions
Evuzamitide
Drug
Lead sponsor
Lorena Saelices
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas
Conditions
Familial Hypercholesterolemia
Interventions
Cascade testing, Standard of Care
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
1 Year and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Gout
Interventions
Arhalofenate 600 mg, Allopurinol 300 mg, Colchicine 0.6 mg, Placebo, Arhalofenate 800 mg
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
52
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Tucson, Arizona + 49 more
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Gout, Hyperuricemia
Interventions
Arhalofenate, Febuxostat, Colchicine
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
Overland Park, Kansas
Completed Early Phase 1 Interventional Accepts healthy volunteers

Apolipoprotein CIII Reduction Via Colchicine

NCT02083510
Conditions
Hypertriglyceridemia, Gout, Pericarditis
Interventions
Colchicine
Drug
Lead sponsor
Scripps Translational Science Institute
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
La Jolla, California
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
Mucopolysaccharidosis II
Interventions
iduronate-2-sulfatase enzyme replacement therapy, Placebo
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
Completed Not applicable Interventional Results available

Cardiac Amyloidosis Discovery Trial

NCT06469372
Conditions
Cardiac Amyloidosis
Interventions
Cardiac amyloidosis deep learning model
Device
Lead sponsor
Pierre Elias
Other
Eligibility
50 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Conditions
Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years and older
Enrollment
151 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1985 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Cidofovir, Probenecid, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
12
States / cities
Los Angeles, California • Miami, Florida • Atlanta, Georgia + 7 more