DDrare: Database of Drug Development for Rare Diseases

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 1-deoxynojirimycin; 1-deoxynojirimycin hydrochloride; A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA); AAV2/8LSPhGAA; ALGLUCOSIDASE ALFA; ALGLUCOSIDASE ALFA (MYOZYME); AT2220; AT2221; AT2221 65 mg Formulated Capsules; AT845; ATB200; Acetaminophen; Albuterol; Aldurazyme (laronidase); Alglucosidase Alfa; Alglucosidase Alfa (Genetical Recombination); Alglucosidase alfa; Alglucosidase alfa (GZ419829); Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]); Alglucosidase alfa GZ419829; Alglucosidase alpha; Alpha-glucosidase; Avalglucosidase Alfa; Avalglucosidase Alfa (NeoGAA); Avalglucosidase alfa; Avalglucosidase alfa GZ402666; Avalglucosidase alfa(GZ402666); BMN 701; Beta-hydroxybuturate esters; Bortezomib; CYCLOPHOSPHAMIDE MONOHYDRATE; Cipaglucosidase alfa; Clenbuterol; Cyclophosphamide; Deoxynojirimycin; Deprakine; Diphenhydramine; Duvoglustat; Duvoglustat HCl; Duvoglustat hydrochloride; Enzyme Replacement Agent; Epilim; Epilim Chrono 200 Controlled Released tablets; Epilim Chrono 300 Controlled Released tablets; Epilim Chrono 500 Controlled Released tablets; Filgrastim; GILT-rhGAA; GZ402666; Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase; Glucosidase alfa; Glycosade; Intravenous immune globulin; Ketocal 4:1 liquid Nutricia (intervention); LMX 4 Topical Cream; Laronidase; Lidocaine; Low carbohydrate ketogenic diet; Lumizyme; Lumizyme®; METHOTREXATE SODIUM; MIGLUSTAT; MYOZYME®; Mannose; Methotrexate; Miglustat; Miglustat (AT2221); Myozyme; Myozyme 50 mg powder for concentrate for solution for infusion; Myozyme®; Myozyme® (alglucosidase alfa); N.a.; NEO-GAA; NeoGAA; NeoGAA or avalglucosidase alfa; Other: No Treatment; Other: RMST; Other: saline; Phosphate; Plecebo; Pyridostigmine; Pyridostigmine Bromide; RAAV1-CMV-GAA (study agent) Administration; RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN; REN001; RITUXIMAB; Rapamycin; ReN001; Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA); Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA); Recombinant human acid alfa-glucosidase; Recombinant human acid alfa-glucosidase (ATB200); Recombinant human acid alfa-glucosidase (rhGAA); Recombinant human acid alpha-glucosidase (rhGAA); Reveglucosidase alfa; Reveglucosidase alpha; RhGAA; RhGAA, acid alpha glucosidase; Rituxan; Rituximab; SODIUM VALPROATE; SPK-3006; Salbutamol; Sodium Valproate; Sodium valproate; Ss-hydroxybuturate esters; TRIHEPTANOIN; Triheptanoin; UX007; VAL-1221; Valproate; Valproic acid; ZAVESCA; Zavesca; Zavesca® Prescription

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2019-003595-38-GB (EUCTR)	20/11/2020	03/07/2020	FORTIS: AAV8-Delivered Gene Therapy in Late Onset Pompe Disease	FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS	Late Onset Pompe Disease MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: AT845 Product Code: AT845	Audentes Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	8	Phase 1;Phase 2	Germany;Italy;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2019-003595-38-GB
(EUCTR)

20/11/2020

03/07/2020

FORTIS: AAV8-Delivered Gene Therapy in Late Onset Pompe Disease

FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS

Late Onset Pompe Disease
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: AT845
Product Code: AT845

Audentes Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

Germany;Italy;United Kingdom