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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: Pompe Disease
Interventions: Clenbuterol, Placebo; Drug
Lead sponsor: Dwight Koeberl, M.D., Ph.D.; Other
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease
Interventions: Pyridostigmine Bromide; Drug
Lead sponsor: University of Florida; Other
Eligibility: 8 Years to 60 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Gainesville, Florida

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: Glycogen Storage Disease, Pompe Disease
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Female only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2024
U.S. locations: 2
States / cities: Cambridge, Massachusetts • Grand Rapids, Michigan

Conditions: Pompe Disease
Interventions: Albuterol, Placebo; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Danon Disease
Interventions: RP-A501; Genetic
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 3
States / cities: La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Pompe Disease
Interventions: duvoglustat, rhGAA; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 11
States / cities: Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more

Conditions: Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 12 Months
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 14
States / cities: Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more

Conditions: Danon Disease
Interventions: RP-A501; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 3
States / cities: La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania

Conditions: Pompe Disease
Interventions: VAL-1221, RhGAA; Drug
Lead sponsor: Valerion Therapeutics, LLC; Industry
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Orange, California • Durham, North Carolina

Conditions: Glycogen Storage Disease
Interventions: Lingual Muscle Training; Other
Lead sponsor: Duke University; Other
Eligibility: 12 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Healthy, Late Onset Pompe Disease
Interventions: ABX1100 injection for IV infusion, Placebo injection for IV infusion; Drug
Lead sponsor: Aro Biotherapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 3
States / cities: Orange, California • St Louis, Missouri • Fairfax, Virginia

Conditions: Pompe Disease, Pregnancy
Interventions: avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666); Biological
Lead sponsor: Sanofi; Industry
Eligibility: Female only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2032
U.S. locations: 1
States / cities: Bridgewater, New Jersey

Conditions: Pompe Disease
Interventions: Rituximab, Miglustat; Drug
Lead sponsor: University of Florida; Other
Eligibility: Up to 65 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2017
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 6 Months and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2010
U.S. locations: 9
States / cities: Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Glycogen Storage Disease Type II
Interventions: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding; Other
Lead sponsor: Columbia University; Other
Eligibility: 8 Years to 17 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: New York, New York

Conditions: Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older · Female only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 1
States / cities: San Antonio, Texas

Conditions: Glycogen Storage Disease Type III
Interventions: UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker; Biological · Other · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 4
States / cities: Orange, California • Atlanta, Georgia • Philadelphia, Pennsylvania + 1 more

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions: There is no intervention; Other
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 64 Years
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2050
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Pompe Disease (Late-onset)
Interventions: zocaglusagene nuzaparvovec; Genetic
Lead sponsor: Astellas Gene Therapies; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2035
U.S. locations: 3
States / cities: Orange, California • Palo Alto, California • Salt Lake City, Utah

Conditions: Pompe Disease
Interventions: respiratory muscle strength training; Other
Lead sponsor: Duke University; Other
Eligibility: 3 Years to 99 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency
Interventions: Observational; Other
Lead sponsor: Duke University; Other
Eligibility: 3 Months to 54 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

Search by objective public record fields.

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

A Pilot Study of Pyridostigmine in Pompe Disease

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

Pompe Lactation Sub-Registry

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Drug-drug Interaction Study

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

Avalglucosidase Alfa Pregnancy Study

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Early Check: Expanded Screening in Newborns

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Gene Transfer Study in Patients With Late Onset Pompe Disease

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease