- Conditions
- Type 1 Gaucher Disease
- Interventions
- Elelyso 60 units/kg
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- Up to 12 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
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 673–696
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Phenylketonuria
- Interventions
- SYNB1934v1, Placebo
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 14
- States / cities
- Culver City, California • Orange, California • Palo Alto, California + 11 more
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- Conditions
- Hypertriglyceridemia
- Interventions
- Omega-3-Acid Ethyl Esters
- Drug
- Lead sponsor
- Jewish Hospital, Cincinnati, Ohio
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Mucopolysaccharidosis I, Cognitive Decline
- Interventions
- Intrathecal recombinant human alpha iduronidase
- Drug
- Lead sponsor
- agnes chen
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Hermansky-Pudlak Syndrome (HPS)
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 115 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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 (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 26
- States / cities
- Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Interventions
- NBI-74788
- Drug
- Lead sponsor
- Neurocrine Biosciences
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Indianapolis, Indiana • Ann Arbor, Michigan + 3 more
- Conditions
- Hyperlipoproteinemia Type I, Hypertriglyceridemia
- Interventions
- Orlistat
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 8 Years to 18 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Cipaglucosidase Alfa, Miglustat
- Biological · Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 9
- States / cities
- Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
- Conditions
- Mucopolysaccharidosis (MPS), Hunter Syndrome
- Interventions
- ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 6 Years · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 8
- States / cities
- Phoenix, Arizona • Oakland, California • San Diego, California + 5 more
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency, Lysosomal Storage Diseases
- Interventions
- Not listed
- Lead sponsor
- Neurogene Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Gout
- Interventions
- Rilonacept, Placebo (for Rilonacept)
- Biological · Other
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 34
- States / cities
- San Diego, California • Santa Maria, California • Whittier, California + 30 more
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin
- Drug
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • Morristown, New Jersey
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- diet
- Behavioral
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Genetic testing
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 378 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 2
- States / cities
- Lancaster, Pennsylvania • Philadelphia, Pennsylvania
- Conditions
- Hemochromatosis
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2003 – 2011
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 24 Weeks to 42 Months
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 5
- States / cities
- Wilmington, Delaware • Springfield, Missouri • Omaha, Nebraska + 2 more
- Conditions
- Phenylketonuria, Maternal
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Female only
- Enrollment
- 50 participants
- Timeline
- 2022 – 2032
- U.S. locations
- 1
- States / cities
- Wilmington, North Carolina
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Zidovudine, Didanosine, Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 3
- States / cities
- Irvine, California • Washington D.C., District of Columbia • Galveston, Texas
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Interventions
- ECUR-506
- Genetic
- Lead sponsor
- iECURE, Inc.
- Industry
- Eligibility
- 24 Hours to 7 Months · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more