- Conditions
- Hunter Syndrome
- Interventions
- Neurobehavioral testing, Visual and auditory assessments
- Behavioral · Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- 32 Months to 18 Years · Male only
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- Chapel Hill, 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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 696
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- DNL126
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 4
- States / cities
- Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
- 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
- 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
- Achondroplasia
- Interventions
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 0 Years to 32 Months
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Oakland, California • Baltimore, Maryland • Madison, Wisconsin
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor, Childhood Cerebral Anaplastic Astrocytoma, Childhood Cerebral Astrocytoma, Childhood Choroid Plexus Neoplasm, Childhood Craniopharyngioma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood Infratentorial Ependymoma, Childhood Oligodendroglioma, Childhood Supratentorial Ependymoma, Ependymoma, Newly Diagnosed Childhood Ependymoma, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Supratentorial Embryonal Tumor, Not Otherwise Specified, Recurrent Childhood Visual Pathway Glioma
- Interventions
- Cytology Specimen Collection Procedure
- Other
- Lead sponsor
- Children's Oncology Group
- Network
- Eligibility
- Up to 18 Years
- Enrollment
- 1,258 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2018
- U.S. locations
- 163
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 123 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Genetic HMI-203
- Biological
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 45 Years · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 5
- States / cities
- Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
- 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
- Type 2 Diabetes, Type II Diabetes Mellitus, Type II Diabetes, Pre-diabetes, Pre-diabetic, Pre-diabetic State, Type 2 Diabetes Mellitus (T2DM), Type 2 Diabetes Mellitus, Type 2 Diabetes (T2DM)
- Interventions
- Marketing Nudges Only, Marketing Nudges + Dynamically Adapted Financial Incentives
- Behavioral
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 45 Years to 70 Years
- Enrollment
- 216 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Gallbladder Cancer, Cholangiocarcinoma, Biliary Tract Cancer
- Interventions
- Not listed
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 390 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Genetic Disease, Inborn Errors of Metabolism, Glycogen Storage Disease, Lysosomal Storage Diseases, Storage Disease
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2,724 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- 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
- Hunter Syndrome
- Interventions
- Idursulfase-IT
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Male only
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 17
- States / cities
- Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
- Conditions
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
- Interventions
- Lactate Meter, Accu Chek
- Other · Device
- Lead sponsor
- Connecticut Children's Medical Center
- Other
- Eligibility
- Up to 60 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Hartford, Connecticut
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II
- Interventions
- Alglucosidase Alfa, Methotrexate, Rituximab
- Biological · Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 2
- States / cities
- Louisville, Kentucky • Durham, North Carolina
- 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
- 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
- Fucosidosis
- Interventions
- Not listed
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 2
- States / cities
- Orange, California • Minneapolis, Minnesota
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 3 Months and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Enriched Hematopoetic Stem Cell Infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- 3 Years and older · Male only
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- Opioid Misuse
- Interventions
- Text Message, Standard of Care
- Other
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 479 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina