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Conditions: Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
Interventions: Not listed
Lead sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); NIH
Eligibility: Not listed
Healthy volunteers: Accepts healthy volunteers
Timeline: 1993 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: Neutralizing Antibody to SPK-3006 capsid; Diagnostic Test
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 6
States / cities: Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more

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: Pompe Disease
Interventions: S-606001, Placebo; Drug
Lead sponsor: Shionogi; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 9
States / cities: Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more

Conditions: Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: Alglucosidase alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003
U.S. locations: 1
States / cities: New Brunswick, New Jersey

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: Pompe Disease
Interventions: rAAV1-CMV-GAA (study agent) Administration, RMST; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 2 Years to 18 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
Interventions: GZ402666; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 7
States / cities: Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more

Conditions: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: SPK-3006; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2032
U.S. locations: 10
States / cities: Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more

Conditions: Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions: AB-1009 (GAA Gene); Genetic
Lead sponsor: AskBio Inc; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 9
States / cities: Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
Interventions: alglucosidase alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 24 Months
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2021
U.S. locations: 3
States / cities: Gainesville, Florida • Decatur, Georgia • Detroit, Michigan

Conditions: Pompe Disease
Interventions: Observational; Other
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2029
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Late-onset Pompe Disease
Interventions: DNL952; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 3
States / cities: Irvine, California • Durham, North Carolina • Fairfax, Virginia

Conditions: Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 6 Months to 36 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 3
States / cities: Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio

Conditions: Pompe Disease
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 5
States / cities: Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more

Conditions: Pompe Disease
Interventions: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream; Genetic · Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 50 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
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: Glycogen Storage Disease Type II
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • St Louis, Missouri • Seattle, Washington

Conditions: Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Female only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2034
U.S. locations: 15
States / cities: Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 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: 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: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: Myozyme® (alglucosidase alfa); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 1 Month and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2020
U.S. locations: 4
States / cities: Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more

Conditions: Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
U.S. locations: 74
States / cities: Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more

Conditions: Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions: Alglucosidase Alfa; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 9
States / cities: Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more

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Genetic and Family Studies of Inherited Muscle Diseases

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Late-Onset Treatment Study Extension Protocol

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

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

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Growth and Development Study of Alglucosidase Alfa

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

High Dose or High Dose Frequency Study of Alglucosidase Alfa

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Pompe Pregnancy Sub-Registry

Early Check: Expanded Screening in Newborns

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.

Immune Tolerance Induction Study

Alglucosidase Alfa Temporary Access Program

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa