- Conditions
- Mucopolysaccharidosis II
- Interventions
- tividenofusp alfa
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 8
- States / cities
- Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
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 567
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Female only
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
- Interventions
- Pyrimethamine
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2008
- U.S. locations
- 2
- States / cities
- New York, New York • Cleveland, Ohio
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Vimizim® (elosulfase alfa)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 418 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 26
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more
- Conditions
- GM1 Gangliosidosis
- Interventions
- LYS-GM101
- Genetic
- Lead sponsor
- LYSOGENE
- Industry
- Eligibility
- Up to 3 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Fabry Disease
- Interventions
- migalastat
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Months and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
- Conditions
- Age-related Macular Degeneration
- Interventions
- Ranibizumab 0.5mg
- Drug
- Lead sponsor
- Southern California Desert Retina Consultants, MC
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 108 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 4
- States / cities
- Mountain View, California • Palm Desert, California • Baltimore, Maryland + 1 more
- Conditions
- Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
- Interventions
- Not listed
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- NIH
- Eligibility
- Not listed
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1993 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Up to 8 Months
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Phoenix, Arizona
- Conditions
- Type I Gaucher Disease
- Interventions
- Ambroxol
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Monmouth Junction, New Jersey
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Tay-Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
- Biological
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Worcester, Massachusetts
- Conditions
- Fabry's Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 325 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1995 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis Type IIIA, Mucopolysaccharidosis Type IIIB
- Interventions
- Lumbar puncture, Magnetic Resonance Imaging (MRI) of the brain
- Procedure · Device
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Gaucher Disease
- Interventions
- Enzyme analysis
- Other
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Sanfilippo Syndrome Type A
- Interventions
- GC1130A
- Drug
- Lead sponsor
- GC Biopharma Corp
- Industry
- Eligibility
- 12 Months to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Gaucher Disease
- Interventions
- Investigational procedure
- Procedure
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- Not listed
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • New York, New York • Milwaukee, Wisconsin
- 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
- Gaucher Disease
- Interventions
- Taliglucerase alfa
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Decatur, Georgia • New York, New York
- 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
- Niemann-Pick Disease, Type C
- Interventions
- IB1001
- Drug
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase Alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more