- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 2
- States / cities
- Oakland, California • Chicago, Illinois
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 1153–1176
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- AEGR-733
- Drug
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Philadelphia, Pennsylvania
- Conditions
- Gout
- Interventions
- pegloticase
- Biological
- Lead sponsor
- Savient Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 151 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 22
- States / cities
- San Francisco, California • Santa Maria, California • Whittier, California + 19 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Roche Global Development
- Other
- Eligibility
- 13 Years and older
- Enrollment
- 280 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 35
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 27 more
- 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
- Homozygous Familial Hypercholesterolemia
- Interventions
- ALN-PCSSC, Standard of Care
- Drug
- Lead sponsor
- The Medicines Company
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Gout, Hyperuricemia, Arthritis, Joint Disease
- Interventions
- ulodesine, Placebo
- Drug
- Lead sponsor
- BioCryst Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 279 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 54
- States / cities
- Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 51 more
- Conditions
- Mild Autonomous Cortisol Secretion, Autonomous Cortisol Secretion (ACS)
- Interventions
- Osilodrostat 1 MG
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Cystinosis
- Interventions
- Safety and Efficacy Assessments
- Other
- Lead sponsor
- Stephanie Cherqui
- Other
- Eligibility
- 14 Years to 50 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2036
- U.S. locations
- 1
- States / cities
- La Jolla, California
- 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
- Metabolism, Inborn Errors, Granulomatous Disease, Chronic
- Interventions
- cyclosporine, fludarabine, mycophenolate mofetil
- Drug
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Chronocort
- Drug
- Lead sponsor
- Immedica Pharma AB
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Orange, California • Jacksonville, Florida + 8 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Eliglustat tartrate, Placebo
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 6
- States / cities
- San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 3 Months to 10 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 6
- States / cities
- Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
- Conditions
- Cystinosis
- Interventions
- Cystagon®, RP103
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Not listed
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Gout
- Interventions
- placebo, pegloticase
- Other · Biological
- Lead sponsor
- Savient Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 50
- States / cities
- Birmingham, Alabama • Tucson, Arizona • Jonesboro, Arkansas + 45 more
- Conditions
- Mucopolysaccharidosis II, MPS II
- Interventions
- SB-913
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Blood Pressure, Gout
- Interventions
- febuxostat
- Drug
- Lead sponsor
- University of Mississippi Medical Center
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Jackson, Mississippi
- 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
- Conditions
- Bile Acid Synthesis Disorders
- Interventions
- Cholbam
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2039
- U.S. locations
- 24
- States / cities
- Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
- Conditions
- Colitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 21 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
- Interventions
- Health-related Quality of Life in Rare Kidney Stone Questionnaire
- Other
- Lead sponsor
- NYU Langone Health
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 365 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2022
- U.S. locations
- 2
- States / cities
- Rochester, Minnesota • New York, New York
- Conditions
- Pancreatic Cancer
- Interventions
- Gemcitabine, TT vaccine booster
- Drug · Biological
- Lead sponsor
- Albert Einstein College of Medicine
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- The Bronx, New York
- Conditions
- Fabry Disease
- Interventions
- AT1001 150 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 4
- States / cities
- Costa Mesa, California • Miami, Florida • Orlando, Florida