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Conditions: Mucopolysaccharidosis III
Interventions: anakinra; Biological
Lead sponsor: Lynda E Polgreen; Other
Eligibility: 4 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Torrance, California

Conditions: Fabry Disease
Interventions: DRX005B; Drug
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Tay-Sachs Disease, Sandhoff Disease
Interventions: venglustat GZ402671, placebo; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 2 Years and older
Enrollment: 75 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2024
U.S. locations: 5
States / cities: Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more

Conditions: Krabbe Disease
Interventions: Not listed
Lead sponsor: University of Pittsburgh; Other
Eligibility: Up to 17 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Fabry Disease, Proteinuria
Interventions: Not listed
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 14 Years to 95 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2006
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
Interventions: Not listed
Lead sponsor: Greenwood Genetic Center; Other
Eligibility: Up to 18 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: Greenville, South Carolina

Conditions: Osteogenesis Imperfecta
Interventions: Risedronate Oral Tablet; Drug
Lead sponsor: Hospital for Special Surgery, New York; Other
Eligibility: 6 Years to 100 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: New York, New York

Conditions: Hunter Syndrome
Interventions: idursulfase-IT, No IT treatment; Biological · Other
Lead sponsor: Shire; Industry
Eligibility: Up to 18 Years · Male only
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina

Conditions: Infantile Globoid Cell Leukodystrophy
Interventions: Not listed
Lead sponsor: Zymenex A/S; Industry
Eligibility: Up to 2 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Fabry's Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 325 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1995 – 2008
U.S. locations: 1
States / cities: Bethesda, Maryland

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: Gaucher Disease
Interventions: Taliglucerase alfa; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 2
States / cities: Decatur, Georgia • New York, New York

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: Gaucher Disease
Interventions: Eliglustat tartrate; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 170 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 10
States / cities: San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more

Conditions: Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions: DUOC-01; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: 1 Week to 22 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Fabry Disease
Interventions: PRX-102; Drug
Lead sponsor: Protalix; Industry
Eligibility: 18 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 9
States / cities: Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more

Conditions: Neuroblastoma, Osteosarcoma
Interventions: iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine; Biological · Drug
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Months and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2044
U.S. locations: 2
States / cities: Atlanta, Georgia • Chapel Hill, North Carolina

Conditions: Pompe Disease, Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: recombinant human acid alpha-glucosidase (rhGAA); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2002
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Morquio A Syndrome, Mucopolysaccharidosis IV A
Interventions: Not listed
Lead sponsor: Nadia Ali, PhD; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Decatur, Georgia

Conditions: Fabry Disease
Interventions: Agalsidase beta; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 5 Years to 18 Years · Male only
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 3
States / cities: Decatur, Georgia • Cincinnati, Ohio • Seattle, Washington

Conditions: Hunter Syndrome
Interventions: Elaprase for intravenous (IV) infusion; Drug
Lead sponsor: Shire; Industry
Eligibility: Up to 5 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Refractive Errors
Interventions: Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens; Device
Lead sponsor: Acuity Polymers, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 74 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2020
U.S. locations: 3
States / cities: Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York

Conditions: Sphingomyelin Lipidosis
Interventions: Olipudase alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 17 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: New York, New York

Conditions: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions: UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy; Biological · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: Not listed
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2029
U.S. locations: 2
States / cities: Columbus, Ohio • Pittsburgh, Pennsylvania

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Open-label Study of Anakinra in MPS III

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Antiproteinuric Agents and Fabry Disease

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

Effects of Bisphosphonates on OI-Related Hearing Loss

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

The Natural History of Infantile Globoid Cell Leukodystrophy

Analysis of the Nervous System in Patients With Fabry's Disease

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH