- Conditions
- Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
- Interventions
- Naglazyme
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 1 Year
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Oakland, California
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 49–72
of 191
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 6 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 3
- States / cities
- Oakland, California • Torrance, California • Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Interventions
- rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2001
- U.S. locations
- 2
- States / cities
- New York, New York • Chapel Hill, North Carolina
- 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
- 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 Type A
- Interventions
- GC1130A
- Drug
- Lead sponsor
- GC Biopharma Corp
- Industry
- Eligibility
- 12 Months to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis II
- Interventions
- tividenofusp alfa
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 8
- States / cities
- Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
- Conditions
- MPS-IH (Hurler Syndrome)
- Interventions
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Genetic
- Lead sponsor
- Orchard Therapeutics
- Industry
- Eligibility
- 28 Days to 30 Months
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome
- Interventions
- Stem Cell Transplant, Laronidase ERT
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 7 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2012
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 236 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
- Interventions
- Pre-visit prep, usual care + exome seq
- Behavioral · Diagnostic Test
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 548 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 3
- States / cities
- Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
- Conditions
- Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
- Interventions
- HGT-1410
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 12 Months to 48 Months
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Chapel Hill, North Carolina
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Hypochondroplasia
- Interventions
- Infigratinib
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2036
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- MGTA-456
- Drug
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Cincinnati, Ohio
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- LYS-SAF302
- Drug
- Lead sponsor
- LYSOGENE
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
- Conditions
- Mucopolysaccharidosis I
- Interventions
- JR-171
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
- Interventions
- FODMAP-targeting digestive enzyme blend, Placebo
- Dietary Supplement · Other
- Lead sponsor
- Kiwi Health Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- 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
- Mucopolysaccharidosis II
- Interventions
- JR-141, Idursulfase, JR-141 or Idursulfase
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more
- Conditions
- Healthy
- Interventions
- Pregabalin, Placebo, Bowel preparation
- Drug · Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Achondroplasia
- Interventions
- Infigratinib
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more