- Conditions
- Mucopolysaccharidosis I
- Interventions
- No Intervention
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Orange, California
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 567
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fabry Disease, Fabry Disease, Cardiac Variant
- Interventions
- ST-920
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2039
- U.S. locations
- 8
- States / cities
- Irvine, California • Tampa, Florida • Atlanta, Georgia + 5 more
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 2
- States / cities
- Oakland, California • Manhasset, New York
- Conditions
- Gaucher Disease
- Interventions
- GA-GCB (velaglucerase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more
- 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
- MPS III B, Mucopolysaccharidosis Type IIIB
- Interventions
- AX 250
- Drug
- Lead sponsor
- Allievex Corporation
- Industry
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Hypochondroplasia
- Interventions
- Not listed
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 30 Months to 16 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
- Conditions
- Gaucher Disease, Type 3
- Interventions
- No intervention
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 5 participants
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Fabry Disease
- Interventions
- PRX-102 (pegunigalsidase alfa)
- Biological
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2021
- U.S. locations
- 5
- States / cities
- Gainesville, Florida • Iowa City, Iowa • Baltimore, Maryland + 2 more
- Conditions
- Melanoma
- Interventions
- Nivolumab, Hydroxychloroquine, Ipilimumab
- Drug
- Lead sponsor
- Ravi Amaravadi, MD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
- Interventions
- Not listed
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Interventions
- Somatropin (DNA origin)
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Parkinson Disease, GBA Gene Mutation, Gaucher Disease
- Interventions
- PET scan, neuroQWERTY
- Diagnostic Test
- Lead sponsor
- Pacific Parkinson's Research Centre
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 2
- States / cities
- Portland, Oregon • Seattle, Washington
- Conditions
- Pompe Disease
- Interventions
- rAAV1-CMV-GAA (study agent) Administration, RMST
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 74
- States / cities
- Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Eliglustat tartrate, Imiglucerase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 17
- States / cities
- Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more
- Conditions
- Gaucher's Disease
- Interventions
- glucocerebrosidase
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Gaucher Disease
- Interventions
- Imiglucerase
- Drug
- Lead sponsor
- University Research Foundation for Lysosomal Storage Diseases, Inc.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 2
- States / cities
- Beverly Hills, California • Coral Springs, Florida
- Conditions
- Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- alglucosidase alfa, Placebo
- Biological · Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 5
- States / cities
- Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
- 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
- Metachromatic Leukodystrophy
- Interventions
- Not listed
- Lead sponsor
- National Organization for Rare Disorders
- Other
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 1
- States / cities
- Danbury, Connecticut
- 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 I
- Interventions
- JR-171
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Oakland, California