- Conditions
- Gaucher's Disease
- Interventions
- Lysodase
- Drug
- Lead sponsor
- National Institute of Mental Health (NIMH)
- NIH
- Eligibility
- Not listed
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Risedronate Oral Tablet
- Drug
- Lead sponsor
- Hospital for Special Surgery, New York
- Other
- Eligibility
- 6 Years to 100 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Fabry Disease
- Interventions
- Replagal
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 4
- States / cities
- Tucson, Arizona • Iowa City, Iowa • New York, New York + 1 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Male only
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 17
- States / cities
- Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
- Conditions
- Achondroplasia
- Interventions
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 0 Years to 32 Months
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Oakland, California • Baltimore, Maryland • Madison, Wisconsin
- 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
- Niemann-Pick Disease, Type C
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Cystinosis
- Interventions
- HIIT Exercise
- Other
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Interventions
- FLT190
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Macular Degeneration
- Interventions
- intravitreal injection Lucentis (ranibizumab)
- Drug
- Lead sponsor
- Retina Research Foundation
- Other
- Eligibility
- 50 Years to 90 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007
- U.S. locations
- 1
- States / cities
- Slingerlands, New York
- Conditions
- Type 2 Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Up to 5 Years · Male only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cerebrovascular Accident, Fabry Disease, Healthy
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1997 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2032
- U.S. locations
- 7
- States / cities
- Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
- Conditions
- Parkinson Disease, GBA Gene Mutation, Gaucher Disease
- Interventions
- PET scan, neuroQWERTY
- Diagnostic Test
- Lead sponsor
- Pacific Parkinson's Research Centre
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 2
- States / cities
- Portland, Oregon • Seattle, Washington
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 74 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 7
- States / cities
- Beverly Hills, California • Coral Springs, Florida • Decatur, Georgia + 4 more
- Conditions
- Gaucher Disease
- Interventions
- Velaglucerase alfa, Vitamin D
- Drug · Dietary Supplement
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 8
- States / cities
- Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
- Conditions
- MPS VI
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa, Placebo
- Biological · Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 125 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 25
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Irvine, California + 21 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV®
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 95 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Oakland, California • Atlanta, Georgia + 7 more
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Interventions
- Adalimumab Injection [Humira], Saline Solution for Injection
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2027
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Lysosomal Storage Disease, Cholesterol Metabolism
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 99 Years
- Enrollment
- 344 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2023
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland