- Conditions
- Wilson Disease, Wilson's Disease, Wilsons Disease
- Interventions
- PM577a
- Drug
- Lead sponsor
- Prime Medicine, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Chicago, Illinois • San Antonio, 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 313–336
of 2,421
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Friedreich Ataxia, Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- Lexeo Therapeutics
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 8
- States / cities
- La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
- Conditions
- Thalassemia
- Interventions
- Zinc Supplementation
- Dietary Supplement
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
- Interventions
- ALN-PCSSC, Normal Saline
- Drug
- Lead sponsor
- The Medicines Company
- Industry
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 501 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 8
- States / cities
- Jacksonville, Florida • Indianapolis, Indiana • New York, New York + 4 more
- Conditions
- Porphyria Cutanea Tarda
- Interventions
- Hydroxychloroquine, Phlebotomy
- Drug · Procedure
- Lead sponsor
- The University of Texas Medical Branch, Galveston
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2021
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, SoC Therapy
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 214 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 6
- States / cities
- Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
- Conditions
- Hyperbilirubinemia, Jaundice
- Interventions
- Gene mutation sample
- Procedure
- Lead sponsor
- Pediatrix
- Other
- Eligibility
- Up to 6 Days
- Enrollment
- 450 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006 – 2007
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- 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
- Phenylketonuria
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 12 Years to 25 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome, Down Syndrome, Epilepsy, Mitochondrial Encephalomyopathies, Cerebral Folate Deficiency
- Interventions
- Not listed
- Lead sponsor
- Richard Frye
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2030
- U.S. locations
- 1
- States / cities
- Phoenix, Arizona
- Conditions
- Cardiac Amyloidosis
- Interventions
- F-18 florbetapir PET
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2018
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Fanconi Anemia
- Interventions
- microarray analysis, biopsy
- Genetic · Procedure
- Lead sponsor
- OHSU Knight Cancer Institute
- Other
- Eligibility
- 1 Year to 55 Years
- Enrollment
- 90 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2002 – 2007
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Parkinson Disease, Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
- Interventions
- Abatacept
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Hyperphagia, Prader-Willi Syndrome
- Interventions
- tDCS
- Device
- Lead sponsor
- University of Kansas Medical Center
- Other
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 31 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 3
- States / cities
- Kansas City, Kansas • Boston, Massachusetts • Dousman, Wisconsin
- Conditions
- Gout
- Interventions
- Joint Biopsy
- Procedure
- Lead sponsor
- Attune Health Research, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Beverly Hills, California
- Conditions
- Porphyria
- Interventions
- heme arginate, tin mesoporphyrin
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1997
- U.S. locations
- 2
- States / cities
- New York, New York • Galveston, Texas
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 12 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 14
- States / cities
- Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
- Conditions
- Sickle Cell Disease, Diabetes Mellitus
- Interventions
- Not listed
- Lead sponsor
- University of Illinois at Chicago
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2029
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Primary Hyperoxaluria
- Interventions
- DCR-PHXC, Placebo
- Drug
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 43 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Cardiac Amyloidosis, Pacemaker
- Interventions
- Pacemaker Rate
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Alpha-Mannosidosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 5 Years to 60 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Abiraterone acetate
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 2 Years to 9 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more