- Conditions
- GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
- Interventions
- IB1001
- Drug
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Rochester, Minnesota • New York, New York
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
- 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
- Pompe Disease
- Interventions
- BMN 701
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 3
- States / cities
- La Jolla, California • Gainesville, Florida • Kansas City, Kansas
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Interventions
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
- Biological · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2029
- U.S. locations
- 2
- States / cities
- Columbus, Ohio • Pittsburgh, Pennsylvania
- Conditions
- Mucopolysaccharidosis Type IIIB
- Interventions
- Not listed
- Lead sponsor
- Allievex Corporation
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2025
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Warfarin
- Drug
- Lead sponsor
- The Cooper Health System
- Other
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Camden, New Jersey
- Conditions
- Bloating
- Interventions
- OPTI-BIOME™ Bacillus subtilis MB40, Placebo
- Dietary Supplement · Other
- Lead sponsor
- BIO-CAT Microbials, LLC
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2017
- U.S. locations
- 2
- States / cities
- Foothill Ranch, California • Orlando, Florida
- Conditions
- Cystinosis
- Interventions
- Cysteamine
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Osteodysplasia
- Interventions
- Marrow stromal cell infusion
- Biological
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2008
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Glycogen Storage Disease Type II
- Interventions
- Myozyme
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 26 Weeks
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 4
- States / cities
- Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- DNL126
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 4
- States / cities
- Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
- Conditions
- Sanfilippo Syndrome Type A
- Interventions
- assessment
- Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- 1 Day to 12 Months
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- 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
- Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Week to 100 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2050
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hunter Syndrome
- Interventions
- Elaprase for intravenous (IV) infusion
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 5 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Gaucher Disease Type 1, Gaucher Disease Type 3
- Interventions
- venglustat (GZ402671), imiglucerase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia
- 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
- 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
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 18 Years · Male only
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
- Conditions
- Fabry Disease
- Interventions
- GZ/SAR402671
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Diagnostic Test
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- Conditions
- SIOD, Cystinosis, FSGS, SLE Nephritis, CKD Stage 4
- Interventions
- Cyclophosphamide 1200 mg/Kg, Fludarabine, Cyclophosphamide 100 mg/Kg, Total Body Irradiation, ATG, Rituximab, Melphalan, CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System, Kidney Transplant
- Drug · Radiation · Device + 1 more
- Lead sponsor
- Alice Bertaina
- Other
- Eligibility
- 1 Year to 30 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2034
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Mucopolysaccharidoses
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 1,147 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 2
- States / cities
- Orange, California • Minneapolis, Minnesota