- Conditions
- Phenylketonuria, PKU
- Interventions
- RDD to Palynziq
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Chevy Chase, Maryland
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 241–260
of 260
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Phenylketonuria
- Interventions
- JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 163 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2025
- U.S. locations
- 11
- States / cities
- Gainesville, Florida • Tampa, Florida • Atlanta, Georgia + 8 more
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Interventions
- AEB1102
- Drug
- Lead sponsor
- Aeglea Biotherapeutics
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 6
- States / cities
- Stanford, California • Gainesville, Florida • Detroit, Michigan + 3 more
- Conditions
- Idiopathic Pulmonary Fibrosis
- Interventions
- No intervention
- Other
- Lead sponsor
- 23andMe, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 690 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Sunnyvale, California
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2033
- U.S. locations
- 12
- States / cities
- Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 13 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
- Interventions
- Ammonia Study Device
- Diagnostic Test
- Lead sponsor
- Sequitur Health Corp.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Valganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Phenylketonurias
- Interventions
- Sapropterin Dihydrochloride, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 6 more
- 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
- Propionic Acidemia
- Interventions
- AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 6 Months to 2 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2033
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Phenylketonuria (PKU)
- Interventions
- BMN165 20mg/day, BMN165 40mg/day, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 215 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 29
- States / cities
- La Jolla, California • Oakland, California • Aurora, Colorado + 26 more
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 14 Days to 20 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1998
- U.S. locations
- 18
- States / cities
- Los Angeles, California • Oakland, California • San Diego, California + 10 more
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Interventions
- Pegzilarginase, Placebo
- Drug
- Lead sponsor
- Immedica Pharma AB
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 17
- States / cities
- Rogers, Arkansas • Orange, California • Stanford, California + 14 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years to 60 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1995
- U.S. locations
- 5
- States / cities
- San Diego, California • San Francisco, California • New York, New York
- Conditions
- Hermansky Pudlak Syndrome
- Interventions
- Pulmonary function test, Chest CT, Sample collection
- Other
- Lead sponsor
- Vanderbilt University
- Other
- Eligibility
- 12 Years to 90 Years
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 5
- States / cities
- Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 more
- Conditions
- OTC Deficiency
- Interventions
- DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2031
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Foscarnet sodium, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 9
- States / cities
- La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
- Conditions
- Homocystinuria
- Interventions
- Pegtibatinase, Placebo
- Drug
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2027
- U.S. locations
- 10
- States / cities
- Aurora, Colorado • Miami, Florida • Chicago, Illinois + 6 more
- Conditions
- Brain Diseases, Metabolic, Inborn, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
- Interventions
- Not listed
- Lead sponsor
- Andrea Gropman
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2026
- U.S. locations
- 12
- States / cities
- Los Angeles, California • Stanford, California • Aurora, Colorado + 9 more