- Conditions
- Pyruvate Dehydrogenase Complex Deficiency Disease
- Interventions
- No intervention
- Other
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2030
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
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 1321–1344
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Kuvan
- Drug
- Lead sponsor
- Dr. Linda Randolph
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2014
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Female only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia
- Interventions
- CTX310
- Drug
- Lead sponsor
- CRISPR Therapeutics AG
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 8
- States / cities
- Hialeah, Florida • Jacksonville, Florida • Orlando, Florida + 5 more
- Conditions
- Mitochondrial Disease
- Interventions
- Diffuse Optical Spectroscopy
- Device
- Lead sponsor
- University of California, Irvine
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Neuronal Ceroid Lipofuscinosis, Batten's Disease
- Interventions
- Not listed
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Behavioral Manifestations of Lesch-Nyhan Disease
- Interventions
- sapropterin
- Drug
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- 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
- 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
- Lysosomal Acid Lipase Deficiency, Wolman Disease
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Palo Alto, California • Minneapolis, Minnesota + 2 more
- Conditions
- Gout
- Interventions
- Pegloticase, methotrexate, placebo, folic acid, gout flare prophylaxis regimen, fexofenadine, acetaminophen, methylprednisolone
- Biological · Drug · Dietary Supplement
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 152 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 60
- States / cities
- Birmingham, Alabama • Anchorage, Alaska • Flagstaff, Arizona + 54 more
- Conditions
- Fabry Disease
- Interventions
- ST-920
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 9
- States / cities
- Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
- Conditions
- Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- Not listed
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Urea Cycle Disorder
- Interventions
- RAVICTI, NaPBA
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Up to 99 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 6
- States / cities
- Gainesville, Florida • New York, New York • Cleveland, Ohio + 3 more
- Conditions
- Gout
- Interventions
- Rilonacept, Placebo
- Biological · Other
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 1,315 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 77
- States / cities
- Alabaster, Alabama • Mesa, Arizona • Burbank, California + 74 more
- Conditions
- Phenylketonurias
- Interventions
- RTX-134
- Drug
- Lead sponsor
- Rubius Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- 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
- Glycogen Storage Disease Type III
- Interventions
- UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker
- Biological · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 4
- States / cities
- Orange, California • Atlanta, Georgia • Philadelphia, Pennsylvania + 1 more
- Conditions
- Recurrent or Tophaceous Gout
- Interventions
- Control, Gout Disease Management Program
- Other
- Lead sponsor
- Kaiser Permanente
- Other
- Eligibility
- 21 Years to 80 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- All KPNC Facilities, California
- Conditions
- CLN7
- Interventions
- AAV9/CLN7
- Genetic
- Lead sponsor
- Benjamin Greenberg
- Other
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD, High Cholesterol
- Interventions
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe, Combination Therapy placebo, Obicetrapib Placebo, Ezetimibe Placebo
- Combination Product · Drug · Other
- Lead sponsor
- NewAmsterdam Pharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 407 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 45
- States / cities
- Gilbert, Arizona • Jacksonville, Florida • Port Orange, Florida + 37 more
- Conditions
- Cholesterol; Lipidosis
- Interventions
- Rosuvastatin 10mg
- Drug
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Other
- Eligibility
- 8 Years to 21 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Kansas City, Missouri
- Conditions
- Sanfilippo Syndrome (MPS III)
- Interventions
- rAAV9-CMV-hNAGLUop
- Biological
- Lead sponsor
- NeuroGT
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2027 – 2029
- U.S. locations
- 1
- States / cities
- St Louis, Missouri