- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 2
- States / cities
- Oakland, California • Philadelphia, Pennsylvania
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 625–648
of 1,511
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fabry Disease
- Interventions
- Replagal (agalsidase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
- Conditions
- Hereditary Amyloidosis, Transthyretin-Related
- Interventions
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Diagnostic Test
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 30 Years to 90 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Familial Hypercholesterolaemia
- Interventions
- Alert-based computerized decision support
- Device
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Amyloidosis
- Interventions
- Patisiran
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 211 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 14
- States / cities
- La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 10 more
- 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
- Mucolipidosis Type IV
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 1 Year to 64 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2021
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
- Interventions
- atumelnant (CRN04894)
- Drug
- Lead sponsor
- Crinetics Pharmaceuticals Inc.
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 3
- States / cities
- Minneapolis, Minnesota • Morehead City, North Carolina • Philadelphia, Pennsylvania
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Abiraterone acetate, Placebo, Hydrocortisone, Fludrocortisone
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 2 Years to 9 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more
- Conditions
- Phenylketonuria
- Interventions
- PTC923
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 247 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 7
- States / cities
- Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
- Conditions
- Hereditary Hemochromatosis
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 21 Years to 100 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Interventions
- BPX-501 and Rimiducid
- Biological
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 4 Months to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
- Interventions
- Venipuncture
- Procedure
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Soft Tissue Sarcoma, Sts, Sarcoma,Soft Tissue
- Interventions
- ADI PEG20, Ifosfamide, Radiotherapy, Mesna
- Drug · Radiation
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Generalised Lipodystrophy, Partial Lipodystrophy
- Interventions
- Metreleptin
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2031
- U.S. locations
- 16
- States / cities
- Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more
- Conditions
- Menkes Disease, Occipital Horn Syndrome
- Interventions
- Droxidopa, Placebo
- Drug · Other
- Lead sponsor
- Stephen G. Kaler, MD
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Evaluation of Plasma Lipoproteins
- Interventions
- Balanced diet
- Behavioral
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 1,017 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1979
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hermansky Pudlak Syndrome, Interstitial Lung Disease
- Interventions
- Pirfenidone
- Drug
- Lead sponsor
- Jesse Roman
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Lysosomal Storage Disease, Cholesterol Metabolism
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 99 Years
- Enrollment
- 344 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2023
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypophosphatasia (HPP)
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Indianapolis, Indiana • St Louis, Missouri + 2 more
- Conditions
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
- Interventions
- enzyme analysis
- Other
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Adrenal Hyperplasia, Congenital
- Interventions
- Not listed
- Lead sponsor
- Office of Rare Diseases (ORD)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 233 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 2
- States / cities
- New York, New York • Dallas, Texas
- Conditions
- Gaucher Disease Type 1
- Interventions
- N-acetylcysteine
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2025
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • New York, New York
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 9,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2034
- U.S. locations
- 91
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more