- 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
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 217–240
of 567
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Minneapolis, Minnesota
- 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
- Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
- Interventions
- Not listed
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- NIH
- Eligibility
- Not listed
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1993 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 3 Months to 10 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 6
- States / cities
- Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
- Conditions
- Niemann Pick Diseases
- Interventions
- OGT918
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2007
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • New York, New York
- 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
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
- Interventions
- Neutralizing Antibody to SPK-3006 capsid
- Diagnostic Test
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Interventions
- RGX-121
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 4 Months to 5 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 4
- States / cities
- Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
- 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
- GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
- Interventions
- Not listed
- Lead sponsor
- Azafaros B.V.
- Industry
- Eligibility
- 2 Years to 20 Years
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 2
- States / cities
- Oakland, California • Rochester, Minnesota
- Conditions
- Leukodystrophy, Globoid Cell
- Interventions
- PBKR03
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 9 Months
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- Digestive Health, Abdominal Discomfort, Gas and Bloating
- Interventions
- Bloat Gummies, Placebo
- Dietary Supplement
- Lead sponsor
- Arrae
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- San Francisco, 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
- Fabry Disease
- Interventions
- Interview
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- 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
- 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
- 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
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- Sanfilippo Syndrome Type C
- Interventions
- Not listed
- Lead sponsor
- Phoenix Nest
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Sanfilippo Syndrome, MPS3
- Interventions
- Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
- Drug
- Lead sponsor
- Ozlem Goker-Alpan
- Other
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis Type IIIA, Mucopolysaccharidosis Type IIIB
- Interventions
- Lumbar puncture, Magnetic Resonance Imaging (MRI) of the brain
- Procedure · Device
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Columbus, Ohio