- Conditions
- Sphingomyelin Lipidosis
- Interventions
- placebo (saline), Olipudase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2023
- U.S. locations
- 3
- States / cities
- San Francisco, California • Decatur, Georgia • The Bronx, New York
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 241–264
of 595
matching trials from the live ClinicalTrials.gov search.
- 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
- Sanfilippo Syndrome (MPS III)
- Interventions
- rAAV9-CMV-hNAGLUop
- Biological
- Lead sponsor
- NeuroGT
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2027 – 2029
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Gaucher Disease
- Interventions
- Digital Engagement Application (GD App), No Intervention
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- recombinant human acid alpha-glucosidase (rhGAA)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2002
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Mucopolysaccharidosis VI
- Interventions
- Placebo, N-acetylgalactosamine 4-sulfatase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2004
- U.S. locations
- 1
- States / cities
- Novato, California
- Conditions
- Niemann-Pick Diseases
- Interventions
- Not listed
- Lead sponsor
- National Niemann-Pick Disease Foundation
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Glycogen Storage Disease
- Interventions
- Medical Food - Glycosade
- Dietary Supplement
- Lead sponsor
- Vitaflo International, Ltd
- Industry
- Eligibility
- 2 Years to 75 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Hartford, Connecticut
- Conditions
- Gaucher Disease, Bone Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 5 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- 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
- 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
- Niemann-Pick Disease, Type C
- Interventions
- arimoclomol, Placebo
- Drug
- Lead sponsor
- ZevraDenmark
- Industry
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2024
- U.S. locations
- 2
- States / cities
- Oakland, California • Rochester, Minnesota
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2008
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Cambridge, Massachusetts
- Conditions
- Irritable Bowel Syndrome
- Interventions
- curcumin, placebo
- Dietary Supplement
- Lead sponsor
- Kaiser Permanente
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 17 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
- Interventions
- GZ402666
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 3
- States / cities
- Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, 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'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
- Hermansky-Pudlak Syndrome (HPS)
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 115 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease, GSD4, GSD IV, APBD
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2035
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Idiopathic Pulmonary Fibrosis
- Interventions
- No intervention
- Other
- Lead sponsor
- 23andMe, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 690 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Sunnyvale, California
- 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