- Conditions
- Hunter Syndrome
- Interventions
- Neurobehavioral testing, Visual and auditory assessments
- Behavioral · Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- 32 Months to 18 Years · Male only
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
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Showing 1–24
of 2,422
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Huntington's Disease
- Interventions
- Pridopidine, Placebo
- Drug · Other
- Lead sponsor
- Prilenia
- Industry
- Eligibility
- 21 Years and older
- Enrollment
- 408 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 16
- States / cities
- La Jolla, California • Los Angeles, California • Englewood, Colorado + 12 more
- Conditions
- LPL Deficiency
- Interventions
- alipogene tiparvovec, Prednisolone, Cyclosporins, Mycophenolate mofetil
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization, Cystic Fibrosis
- Interventions
- AZLI, Placebo
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 3 Months to 18 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 12
- States / cities
- San Francisco, California • Washington D.C., District of Columbia • St. Petersburg, Florida + 8 more
- Conditions
- Cystic Fibrosis, Inflammatory Bowel Disease, Type 1 Diabetes, Chronic Disease Management
- Interventions
- MD2ME
- Behavioral
- Lead sponsor
- Huang, Jeannie, M.D.
- Individual
- Eligibility
- 14 Years to 22 Years
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Hereditary Angioedema
- Interventions
- Icatibant
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 5
- States / cities
- La Jolla, California • Los Angeles, California • Wheaton, Maryland + 2 more
- Conditions
- Down Syndrome, Cognitive Dysfunction
- Interventions
- Aricept (donepezil hydrochloride), Placebo
- Drug
- Lead sponsor
- Eisai Inc.
- Industry
- Eligibility
- 11 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008
- U.S. locations
- 2
- States / cities
- San Antonio, Texas • Herndon, Virginia
- Conditions
- Age-related Macular Degeneration
- Interventions
- RetnaGene AMD LDT
- Device
- Lead sponsor
- Sequenom, Inc.
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 3
- States / cities
- San Francisco, California • Lexington, Kentucky • Nashville, Tennessee
- Conditions
- Huntington's Disease
- Interventions
- Not listed
- Lead sponsor
- University College, London
- Other
- Eligibility
- 11 Years to 75 Years
- Enrollment
- 2,500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2017
- U.S. locations
- 6
- States / cities
- Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
- Interventions
- Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
- Biological
- Lead sponsor
- AC Immune SA
- Industry
- Eligibility
- 35 Years to 85 Years
- Enrollment
- 304 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
- Conditions
- Advanced Solid Tumors
- Interventions
- BMS-986484, Nivolumab, Oxaliplatin, Capecitabine, Fluorouracil, Calcium folinate
- Drug · Biological
- Lead sponsor
- Bristol-Myers Squibb
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 213 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 5
- States / cities
- Tucson, Arizona • Los Angeles, California • Grand Rapids, Michigan + 2 more
- Conditions
- Leukemia, Lymphoma
- Interventions
- Not listed
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 137 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2024
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Tourette Syndrome
- Interventions
- Valbenazine, Placebo oral capsule
- Drug
- Lead sponsor
- Neurocrine Biosciences
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 127 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2018
- U.S. locations
- 34
- States / cities
- Sun City, Arizona • Rogers, Arkansas • Anaheim, California + 27 more
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, Buphenyl (NaPBA)
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 21
- States / cities
- Long Beach, California • Los Angeles, California • Stanford, California + 18 more
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
- Interventions
- EDIT-101
- Drug
- Lead sponsor
- Editas Medicine, Inc.
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 5
- States / cities
- Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Interventions
- turoctocog alfa
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 6 Months to 70 Years · Male only
- Enrollment
- 214 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Long Beach, California • Tampa, Florida + 10 more
- Conditions
- Adrenoleukodystrophy
- Interventions
- MIN-102, Placebos
- Drug
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2025
- U.S. locations
- 3
- States / cities
- Stanford, California • Baltimore, Maryland • Boston, Massachusetts
- Conditions
- JOB's Syndrome, Hyper-IgE Recurrent Infection Syndrome, Immune Deficiency
- Interventions
- Ranitidine, Placebo
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 2 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis
- Interventions
- Mycophenolate mofetil, Liquid Placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Gout, Hyperuricemia
- Interventions
- Arhalofenate, Febuxostat, Colchicine
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Recurrent or Tophaceous Gout
- Interventions
- Control, Gout Disease Management Program
- Other
- Lead sponsor
- Kaiser Permanente
- Other
- Eligibility
- 21 Years to 80 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- All KPNC Facilities, California
- Conditions
- Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- Nicole Hamblett
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 490 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 56
- States / cities
- Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 47 more
- Conditions
- Hemophilia, Thrombosis, Hemophilia A, Hemophilia B, Sickle Cell Disease, Glanzmann Thrombasthenia, Bleeding Disorder, Blood Disorder, Von Willebrand Diseases
- Interventions
- Not listed
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Network
- Eligibility
- Not listed
- Enrollment
- 200,000 participants
- Timeline
- 2024 – 2055
- U.S. locations
- 1
- States / cities
- Hickory, North Carolina