- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Timeline
- 2017 – 2028
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
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 433–456
of 567
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fabry Disease
- Interventions
- A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Gaucher Disease, Type 2
- Interventions
- LY3884961, Methylprednisolone, Sirolimus, Prednisone
- Genetic · Drug
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 0 Months to 24 Months
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 5
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more
- Conditions
- Gaucher Disease
- Interventions
- Standard of Care
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- 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
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Female only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Sanfilippo Syndrome Type A (MPS IIIA)
- Interventions
- SOBI003
- Drug
- Lead sponsor
- Swedish Orphan Biovitrum
- Industry
- Eligibility
- 18 Months to 78 Months
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 2
- States / cities
- Oakland, California • Chapel Hill, North Carolina
- 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
- Fabry Disease
- Interventions
- Agalsidase alfa
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2011
- U.S. locations
- 13
- States / cities
- Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more
- Conditions
- Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
- Interventions
- Not listed
- Lead sponsor
- Cystinosis Research Foundation
- Other
- Eligibility
- Not listed
- Enrollment
- 750 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2022
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl Cysteine
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 12 Months and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Cystinosis
- Interventions
- Cysteamine Bitartrate Delayed-release Capsules
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2017
- U.S. locations
- 5
- States / cities
- San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
- Conditions
- Fabry Disease
- Interventions
- PRX-102 1 mg/kg every two weeks
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2031
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Atlanta, Georgia • Iowa City, Iowa + 3 more
- 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
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
- Interventions
- Venipuncture
- Procedure
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Pseudotumor Cerebri, Cystinosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Not listed
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Fabry Disease
- Interventions
- Agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 6
- States / cities
- Coral Springs, Florida • Decatur, Georgia • Baltimore, Maryland + 3 more
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2034
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 more
- Conditions
- Gaucher's Disease, Osteopenia
- Interventions
- alendronate sodium, calcium carbonate, cholecalciferol
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 82 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2002
- U.S. locations
- 2
- States / cities
- Cincinnati, Ohio • Dayton, Ohio
- Conditions
- Fabry Disease
- Interventions
- DRX005B
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 7 Years to 15 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 1
- States / cities
- Tucson, Arizona