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 ... show all
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	EUCTR2019-000954-67-AT (EUCTR)	19/06/2020	13/03/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Other descriptive name: AT2221 65 mg Formulated Capsules	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden

No.

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Public_title

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Condition

Intervention

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Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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EUCTR2019-000954-67-AT
(EUCTR)

19/06/2020

13/03/2020

A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease

Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Other descriptive name: AT2221 65 mg Formulated Capsules

Amicus Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

110

Phase 3

United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden