- Conditions
- Phenylketonuria, Galactosemia, Inborn Errors of Metabolism
- Interventions
- Not listed
- Lead sponsor
- University of Miami
- Other
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Miami, Florida
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 505–528
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Amino Acid Metabolism, Inborn Errors
- Interventions
- Protein and calorie controlled diet, Ornithine transcarbamylase vector
- Behavioral · Genetic
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 6 Months to 64 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Phenylketonuria
- Interventions
- Pegvaliase, Methotrexate
- Biological · Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 12
- States / cities
- Los Angeles, California • Aurora, Colorado • Tampa, Florida + 9 more
- Conditions
- Glycogen Storage Disease Type II
- Interventions
- High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
- Other
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 8 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Tafamidis, Placebo
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 441 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 50
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
- Conditions
- Cardiac Amyloidosis
- Interventions
- AI ECG Amyloid algorithm
- Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20,143 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 4
- States / cities
- Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 1 more
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Interventions
- AR882 Dose 1, AR882 Dose 2, Allopurinol Tablet
- Drug
- Lead sponsor
- Arthrosi Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 10
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Sun City, Arizona + 7 more
- Conditions
- 21-hydroxylase Deficiency
- Interventions
- Hydrocortisone withdrawal
- Procedure
- Lead sponsor
- Maria I. New
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 2
- States / cities
- New York, New York • Dallas, Texas
- Conditions
- Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2027
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Danon Disease
- Interventions
- RP-A501
- Genetic
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older · Male only
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 3
- States / cities
- La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
- Conditions
- Undiagnosed Disease, Healthy Volunteer (Adult With Typically Developing Child), Inherited Neurodegenerative Disorders, Inherited Metabolic Disorders (Inborn Errors of Metabolism), Alzheimer's Disease and Related Dementias
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 682 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2012
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Memphis, Tennessee
- Conditions
- Gastrointestinal, Colic
- Interventions
- Lactobacillus reuteri, Placebo
- Biological
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- 3 Weeks to 3 Months
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Batten Disease, CLN6
- Interventions
- Natural history
- Other
- Lead sponsor
- Emily de los Reyes
- Other
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Propionic Acidemia, Methylmalonic Acidemia
- Interventions
- N-carbamylglutamate, Standard of Care
- Drug · Other
- Lead sponsor
- Mendel Tuchman
- Other
- Eligibility
- 1 Hour to 4 Weeks
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Palo Alto, California • Aurora, Colorado + 4 more
- Conditions
- Phenylketonurias
- Interventions
- Skin Tape, Blood Draw
- Other · Diagnostic Test
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- Not listed
- Enrollment
- 86 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2021
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
- Interventions
- acoramidis, Placebo Oral Tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 632 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 40
- States / cities
- Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
- 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
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2009
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Phenylketonuria
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 111 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Sacramento, California • San Jose, California + 11 more
- Conditions
- Mucolipidosis Type IV
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- X-linked Hypophosphatemia
- Interventions
- KRN23
- Drug
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- MELAS Syndrome
- Interventions
- Fluzone®
- Biological
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 13 Years to 65 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2012
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Gout, Hyperuricemia
- Interventions
- ABP-671, Placebo
- Drug · Other
- Lead sponsor
- Atom Therapeutics Co., Ltd
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Tempe, Arizona