- Conditions
- Phenylketonuria (PKU) and Hyperphenylalaninemia
- Interventions
- PheCheck, Dried Blood Spot Cards.
- Device · Diagnostic Test
- Lead sponsor
- Aptatek BioSciences, Inc
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Boston, Massachusetts
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 337–360
of 1,709
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Homocystinuria
- Interventions
- taurine
- Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 8 Years to 49 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2017
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Durham, North Carolina • Philadelphia, Pennsylvania
- Conditions
- Sjogren-Larsson Syndrome (SLS)
- Interventions
- Not listed
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 2
- States / cities
- Omaha, Nebraska • Pittsburgh, Pennsylvania
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
- Interventions
- Parent-Infant Inter(X)action Intervention (PIXI)
- Behavioral
- Lead sponsor
- RTI International
- Other
- Eligibility
- Up to 99 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Friedreich Ataxia
- Interventions
- CTI-1601
- Drug
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 2 Years to 60 Years
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 11
- States / cities
- Los Angeles, California • San Francisco, California • Gainesville, Florida + 8 more
- Conditions
- Tourette Syndrome, Tic Disorder
- Interventions
- This study involves no intervention.
- Other
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 11 Years to 17 Years
- Enrollment
- 351 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2030
- U.S. locations
- 1
- States / cities
- Nashvile, Tennessee
- Conditions
- Lennox Gastaut Syndrome
- Interventions
- Carisbamate
- Drug
- Lead sponsor
- SK Life Science, Inc.
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 7
- States / cities
- Baltimore, Maryland • Lebanon, New Hampshire • Durham, North Carolina + 4 more
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma
- Interventions
- amifostine trihydrate, busulfan, filgrastim, melphalan, thiotepa, peripheral blood stem cell transplantation (PBSC)
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 1 Year to 45 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2003
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Cerebral Cavernous Malformations
- Interventions
- Propranolol, DNA and RNA Analysis
- Drug · Genetic
- Lead sponsor
- St. Joseph's Hospital and Medical Center, Phoenix
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 1
- States / cities
- Phoenix, Arizona
- Conditions
- Phenylketonuria, Hyperphenylalaninaemia
- Interventions
- Kuvan
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,887 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2021
- U.S. locations
- 45
- States / cities
- Los Angeles, California • Orange, California • San Diego, California + 38 more
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Interventions
- Sonlicromanol, Placebo
- Drug
- Lead sponsor
- Khondrion BV
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 220 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
- Conditions
- Gaucher Disease, Bone Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 5 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Neural Tube Defects, Contraception, Oral Contraceptives (OC)
- Interventions
- Drospirenone/Ethinylestradiol/Methyltetrahydrofolate, Drospirenone/Ethinylestradiol (Yaz)
- Drug
- Lead sponsor
- Bayer
- Industry
- Eligibility
- 18 Years to 40 Years · Female only
- Enrollment
- 385 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2008
- U.S. locations
- 9
- States / cities
- Anaheim, California • San Diego, California • Baltimore, Maryland + 6 more
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10
- Interventions
- troriluzole, Placebo
- Drug
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 299 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 21
- States / cities
- Phoenix, Arizona • Long Beach, California • Los Angeles, California + 16 more
- Conditions
- Fabry Disease
- Interventions
- Venglustat (GZ402671), Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Los Angeles, California • Orange, California + 8 more
- Conditions
- Hyper IgD Syndrome, Mevalonate Kinase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Michigan Technological University
- Other
- Eligibility
- 18 Years to 89 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 3
- States / cities
- Houghton, Michigan • Omaha, Nebraska • Portland, Oregon
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, RFC1 Gene Mutation, Spinocerebellar Ataxia Type 27b, Healthy Participants
- Interventions
- Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan, Assessments and Questionnaires, Cerebrospinal Fluid Collection
- Genetic · Other
- Lead sponsor
- Lauren Moore
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 1,400 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2030
- U.S. locations
- 16
- States / cities
- Los Angeles, California • San Francisco, California • Gainesville, Florida + 12 more
- Conditions
- Rett Syndrome
- Interventions
- fenfluramine hydrochloride, Placebo
- Drug · Other
- Lead sponsor
- UCB BIOSCIENCES, Inc.
- Industry
- Eligibility
- 5 Years to 35 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 8
- States / cities
- Little Rock, Arkansas • Miami, Florida • Orlando, Florida + 5 more
- Conditions
- Neurofibromatosis Type II
- Interventions
- Everolimus (RAD001) , Afinitor®
- Drug
- Lead sponsor
- NYU Langone Health
- Other
- Eligibility
- 3 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Tourette Syndrome
- Interventions
- Modified Atkins diet
- Dietary Supplement
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Interventions
- Acoramidis (AG10) oral tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- 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
- Tourette Syndrome, Tic Disorders
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 21 Years to 65 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2003 – 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Friedreich Ataxia
- Interventions
- Not listed
- Lead sponsor
- Friedreich's Ataxia Research Alliance
- Other
- Eligibility
- Not listed
- Enrollment
- 3,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2048
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more