- Conditions
- Phenylketonuria
- Interventions
- AG-181
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- Indianapolis, Indiana • Pittsburgh, Pennsylvania • Dallas, Texas
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 385–408
of 1,709
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Huntington Disease
- Interventions
- SPK-10001, Placebo Surgery Control
- Genetic · Other
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 25 Years to 65 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2035
- U.S. locations
- 5
- States / cities
- Boston, Massachusetts • Cincinnati, Ohio • Columbus, Ohio + 2 more
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Intracranial Hemorrhages, Leukomalacia, Periventricular, Cerebral Palsy
- Interventions
- Not listed
- Lead sponsor
- NICHD Neonatal Research Network
- Network
- Eligibility
- Up to 14 Days
- Enrollment
- 1,339 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1996 – 2000
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Palo Alto, California • New Haven, Connecticut + 12 more
- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 1
- States / cities
- North Charleston, South Carolina
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- SRP-4045, SRP-4053, Placebo
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 13 Years · Male only
- Enrollment
- 228 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2025
- U.S. locations
- 23
- States / cities
- Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Angelman Syndrome
- Interventions
- OV101
- Drug
- Lead sponsor
- Healx AI
- Industry
- Eligibility
- 13 Years to 49 Years
- Enrollment
- 141 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • San Diego, California • Atlanta, Georgia + 7 more
- Conditions
- Age-Related Macular Degeneration, Macular Degeneration
- Interventions
- Intravitreal Aflibercept Injection
- Drug
- Lead sponsor
- MidAtlantic Retina
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 3
- States / cities
- Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
- 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
- Krabbe Disease
- Interventions
- Not listed
- Lead sponsor
- State University of New York at Buffalo
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2026
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Alcohol Dependence
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 18 Years to 60 Years · Male only
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Cerebral Cavernous Malformation
- Interventions
- REC-994, Placebo
- Drug
- Lead sponsor
- Recursion Pharmaceuticals Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 11 more
- Conditions
- Alzheimer Disease, Down Syndrome
- Interventions
- ION269
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 35 Years to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 5
- States / cities
- Indianapolis, Indiana • Kansas City, Kansas • Lexington, Kentucky + 2 more
- Conditions
- Lysosomal Storage Diseases
- Interventions
- Enzyme assay and molecular sequencing
- Diagnostic Test
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 100,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Friedreich Ataxia
- Interventions
- CTI-1601, Placebo
- Biological · Other
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Cancer
- Interventions
- Fentanyl sublingual spray, Placebo
- Drug
- Lead sponsor
- INSYS Therapeutics Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 130 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Chandler, Arizona
- Conditions
- Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- Alglucosidase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003
- U.S. locations
- 1
- States / cities
- New Brunswick, New Jersey
- Conditions
- Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder, Autism Spectrum Disorder High-Functioning, Alzheimer Disease, Alzheimer Dementia (AD), Alzheimer Dementia, Alzheimer Disease (AD), Crohn, Crohn Colitis, Crohn Disease (CD), Crohn Disease Colon, Myalgic Encephalomyelitis, ME/CFS, Myalgic Encephalomyelitis (ME), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome, Psoriasis, Psoriasis Annularis, Psoriasis Chronic, Chronic Urinary Tract Infection, Ulcerative Colitis (Disorder), Ulcerative Colitis (UC), Ulcerative Colitis Acute, Ulcerative Colitis Chronic, Multiple Sclerosis, Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (MS) Primary Progressive, Constipation Chronic Idiopathic, Constipation, Celiac, Celiac Disease, Celiac Sprue, Lyme Arthritis, Lyme Borreliosis, Nervous System, Lyme Disease, Chronic, Cholesterol, Cholesterol Level, High, Cancer, Colon Cancer, Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis, Rheumatoid Arthritis (RA), Rheumatoid Arthritis - Rheumatism, Chronic Fatigue Syndrome (CFS), PARKINSON DISEASE (Disorder), Parkinson Disease, Parkinson Disease (PD), Depression, Major Depressive Disorder (MDD), Depression Disorder, Depression in Adults, Anxiety, Anxiety Disorder Generalized, Obsessive Compulsive Disorder (OCD), Obsessive Compulsive Disorder OCD, Obsessive - Compulsive Disorder, Bipolar, Bipolar 1 Disorder, Bipolar Disorder (BD), Bipolar Disorder I and II, Bipolar and Related Disorders, Migraine, Migraine Disorder, Diabetes (DM), Diabetes, Lupus, Lupus Erythematosus, Epidermolysis Bullosa (EB), Mesothelioma, Mesothelioma Malignant, IBS (Irritable Bowel Syndrome), Irritable Bowel, IBS - Irritable Bowel Syndrome, Irritable Bowel Syndrome (IBS), Eczema, Eczema Atopic Dermatitis, Acne, Myasthaenia Gravis, Gout
- Interventions
- There is no intervention in this study
- Other
- Lead sponsor
- ProgenaBiome
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2030
- U.S. locations
- 1
- States / cities
- Ventura, California
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Mitochondrial Dysfunction, Genetic Disease
- Interventions
- ATI-105, Placebo
- Drug
- Lead sponsor
- Adjuvia Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Huntington Disease
- Interventions
- RO7234292 (RG6042)
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 25 Years and older
- Enrollment
- 236 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • La Jolla, California • Palo Alto, California + 11 more
- Conditions
- Tourette's Syndrome
- Interventions
- PF-03654746, Placebo
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 55 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012
- U.S. locations
- 1
- States / cities
- Manhasset, New York
- Conditions
- Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
- Interventions
- LNP.UCD.ABE
- Biological
- Lead sponsor
- Rebecca Ahrens-Nicklas
- Other
- Eligibility
- 24 Hours to 5 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Friedreich's Ataxia
- Interventions
- Aerobic Exercise
- Behavioral
- Lead sponsor
- Scott Barbuto
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Ocular Motility Disorders, Strabismus, Extraocular Muscle Dysfunction, Thyroid Eye Disease
- Interventions
- Glandy EOM (AI-based quantitative eye movement analysis software)
- Device
- Lead sponsor
- THYROSCOPE INC.
- Industry
- Eligibility
- 22 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- San Jose, California