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	Countries
1	NCT04327973 (ClinicalTrials.gov)	September 1, 2021	27/3/2020	Expanded Access for ATB200/AT2221 for the Treatment of IOPD	Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease	Pompe Disease Infantile-Onset	Biological: ATB200;Drug: AT2221	Amicus Therapeutics	NULL	Available	N/A	17 Years	All		United States;Italy;Taiwan
2	EUCTR2011-002154-32-GB (EUCTR)	07/12/2011	07/10/2011	Drug-drug interaction study between AT2220 and ERT	AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE	Pompe Disease MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: Duvoglustat hydrochloride Product Code: AT2220 INN or Proposed INN: Duvoglustat hydrochloride Other descriptive name: 1-deoxynojirimycin hydrochloride Product Name: Duvoglustat hydrochloride Product Code: AT2220 INN or Proposed INN: Duvoglustat hydrochloride Other descriptive name: 1-deoxynojirimycin hydrochloride	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	22	United States;France;Canada;United Kingdom
3	EUCTR2008-002302-18-DE (EUCTR)	16/02/2009	26/09/2008		An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease -	Pompe Disease MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease	Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	United Kingdom;Germany
4	EUCTR2008-002302-18-GB (EUCTR)	18/12/2008	07/01/2009		An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease -	Pompe Disease MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease	Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Germany;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

NCT04327973
(ClinicalTrials.gov)

September 1, 2021

27/3/2020

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease

Pompe Disease Infantile-Onset

Biological: ATB200;Drug: AT2221

Amicus Therapeutics

NULL

Available

N/A

17 Years

All

United States;Italy;Taiwan

EUCTR2011-002154-32-GB
(EUCTR)

07/12/2011

07/10/2011

Drug-drug interaction study between AT2220 and ERT

AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE

Pompe Disease
MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: Duvoglustat hydrochloride
Product Code: AT2220
INN or Proposed INN: Duvoglustat hydrochloride
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Name: Duvoglustat hydrochloride
Product Code: AT2220
INN or Proposed INN: Duvoglustat hydrochloride
Other descriptive name: 1-deoxynojirimycin hydrochloride

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United States;France;Canada;United Kingdom

EUCTR2008-002302-18-DE
(EUCTR)

16/02/2009

26/09/2008

An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease -

Pompe Disease
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease

Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United Kingdom;Germany

EUCTR2008-002302-18-GB
(EUCTR)

18/12/2008

07/01/2009

An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease -

Pompe Disease
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease

Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Germany;United Kingdom