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	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	NCT01380743 (ClinicalTrials.gov)	October 31, 2011	23/6/2011	Drug-drug Interaction Study	An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease	Pompe Disease	Drug: duvoglustat;Drug: rhGAA	Amicus Therapeutics	NULL	Completed	18 Years	65 Years	All	25	Phase 2	United States;Canada;France;United Kingdom
4	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
5	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
6	NCT00688597 (ClinicalTrials.gov)	December 8, 2008	30/5/2008	Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease	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	Drug: Duvoglustat	Amicus Therapeutics	NULL	Terminated	18 Years	74 Years	All	3	Phase 2	United States;Australia;Canada;France;Germany;Netherlands;United Kingdom
7	EUCTR2013-002257-30-GB (EUCTR)		21/10/2013	A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients	AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE	Pompe disease MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	24	Phase 2	United States;Netherlands;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

NCT01380743
(ClinicalTrials.gov)

October 31, 2011

23/6/2011

Drug-drug Interaction Study

An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease

Pompe Disease

Drug: duvoglustat;Drug: rhGAA

Amicus Therapeutics

NULL

Completed

18 Years

65 Years

All

Phase 2

United States;Canada;France;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

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

NCT00688597
(ClinicalTrials.gov)

December 8, 2008

30/5/2008

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

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

Drug: Duvoglustat

Amicus Therapeutics

NULL

Terminated

18 Years

74 Years

All

Phase 2

United States;Australia;Canada;France;Germany;Netherlands;United Kingdom

EUCTR2013-002257-30-GB
(EUCTR)

21/10/2013

A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients

AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE

Pompe disease
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Netherlands;United Kingdom