- Conditions
- Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 5,000 participants
- Timeline
- 2016 – 2090
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • Cincinnati, Ohio
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 769–792
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Interventions
- GEB-101 Injection
- Genetic
- Lead sponsor
- GenEditBio Limited
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
- Interventions
- ALDHbr Umbilical Cord Blood Cells
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2012
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Amyloidosis Cardiac
- Interventions
- Sonotherapy
- Combination Product
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Gout
- Interventions
- Lesinurad, Placebo, Allopurinol
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 610 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 88
- States / cities
- Mobile, Alabama • Chandler, Arizona • Mesa, Arizona + 76 more
- Conditions
- Sanfilippo Syndrome, Mucopolysaccharidosis III
- Interventions
- Epidiolex, Placebo
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Schwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Fanconi Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia
- Interventions
- Alefacept
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Pompe Disease
- Interventions
- Albuterol
- Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hypocalcemia, Pseudohypoparathyroidism, Osteomalacia, Rickets, Pseudopseudohpoparathyroidism
- Interventions
- Not listed
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- 2 Months to 100 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Phenylketonuria
- Interventions
- Kuvan
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 6 Years to 18 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Mild Cognitive Impairment
- Interventions
- Not listed
- Lead sponsor
- Pamlab, Inc.
- Industry
- Eligibility
- 55 Years to 89 Years
- Enrollment
- 700 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2014
- U.S. locations
- 1
- States / cities
- Springfield, Massachusetts
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Interventions
- Crinecerfont
- Drug
- Lead sponsor
- Neurocrine Biosciences
- Industry
- Eligibility
- 14 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 6
- States / cities
- San Diego, California • Aurora, Colorado • Ann Arbor, Michigan + 3 more
- Conditions
- Fabry Disease
- Interventions
- Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
- Diagnostic Test
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
- Interventions
- Group 1
- Other
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 8 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Safety and Pharmacokinetics in Healthy Volunteer Subjects
- Interventions
- CC-223
- Drug
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Mitochondrial Disease
- Interventions
- Diffuse Optical Spectroscopy
- Device
- Lead sponsor
- University of California, Irvine
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Gout
- Interventions
- DECT scan
- Procedure
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 223 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 9
- States / cities
- Irvine, California • Englewood, Colorado • Wheat Ridge, Colorado + 6 more
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- No Intervention
- Other
- Lead sponsor
- Astellas Gene Therapies
- Industry
- Eligibility
- 16 Years to 69 Years
- Enrollment
- 119 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 10
- States / cities
- Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more
- 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
- Niemann-Pick Disease, Type C
- Interventions
- Adrabetadex
- Drug
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
- Conditions
- Phenylketonuria
- Interventions
- rAvPAL-PEG
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 50 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 8
- States / cities
- Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more