- Conditions
- Alpha-Mannosidosis
- Interventions
- Velmanase Alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Up to 3 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 2
- States / cities
- Miami, Florida • Greenwood, South Carolina
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.
Local D1 index available.
- Conditions
- Age-Related Macular Degeneration, Macular Degeneration
- Interventions
- Intravitreal Aflibercept Injection
- Drug
- Lead sponsor
- MidAtlantic Retina
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 3
- States / cities
- Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
- Conditions
- Morquio Disease, MPS - Mucopolysaccharidosis
- Interventions
- Interview
- Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 15 Years to 100 Years · Female only
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, agalsidase
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
- Conditions
- Cystinosis
- Interventions
- Cysteamine
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 2 Years and older
- Enrollment
- 328 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1986 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin
- Drug
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • Morristown, New Jersey
- Conditions
- Fabry Disease
- Interventions
- agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
- Conditions
- MPS III B
- Interventions
- AX 250
- Drug
- Lead sponsor
- Allievex Corporation
- Industry
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 2
- States / cities
- Oakland, California • Pittsburgh, Pennsylvania
- Conditions
- Gas and Bloating, Diarrhea, Constipation
- Interventions
- GI Health Active Product 1, GI Health Product Placebo Control
- Dietary Supplement
- Lead sponsor
- Radicle Science
- Industry
- Eligibility
- 21 Years to 105 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Del Mar, California
- Conditions
- Lysosomal Storage Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl-L-Leucine, Placebo
- Drug · Other
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 1 Year to 75 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2030
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota
- Conditions
- Glycogen Storage Disease Type II
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2006
- U.S. locations
- 3
- States / cities
- Washington D.C., District of Columbia • St Louis, Missouri • Seattle, Washington
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
- Interventions
- Cerezyme (imiglucerase for injection)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 10 Years to 65 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2004
- U.S. locations
- 1
- States / cities
- Coral Springs, Florida
- Conditions
- Mucopolysaccharidosis Type IVA
- Interventions
- Not listed
- Lead sponsor
- Emory University
- Other
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Fabry Disease
- Interventions
- pegunigalsidase alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Clenbuterol, Placebos
- Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Fabry's Disease
- Interventions
- Smartpill, Endoscopic Mucosal Resection
- Device · Procedure
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase-alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 7 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
- Interventions
- HGT-1410
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 12 Months to 48 Months
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Chapel Hill, North Carolina
- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141, Idursulfase, JR-141 or Idursulfase
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more