DDrare: Database of Drug Development for Rare Diseases

256. 筋型糖原病
［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］

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	NCT02405598 (ClinicalTrials.gov)	October 2013	22/3/2015	Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease	Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease	Pompe Disease	Drug: Salbutamol	National Taiwan University Hospital	NULL	Completed	2 Years	N/A	Both	14	Phase 4	Taiwan
2	NCT01885936 (ClinicalTrials.gov)	June 2013	21/6/2013	Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease	A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy	Pompe Disease	Drug: Albuterol;Drug: Placebo	Duke University	NULL	Completed	18 Years	N/A	All	16	Phase 1;Phase 2	United States
3	NCT01859624 (ClinicalTrials.gov)	June 2012	8/5/2013	Albuterol in Individuals With Late Onset Pompe Disease (LOPD)	A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement Therapy	Pompe Disease	Drug: Albuterol	Duke University	NULL	Completed	18 Years	N/A	All	8	Phase 1	United States

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

NCT02405598
(ClinicalTrials.gov)

October 2013

22/3/2015

Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

Pompe Disease

Drug: Salbutamol

National Taiwan University Hospital

NULL

Completed

2 Years

N/A

Both

Phase 4

Taiwan

NCT01885936
(ClinicalTrials.gov)

June 2013

21/6/2013

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy

Pompe Disease

Drug: Albuterol;Drug: Placebo

Duke University

NULL

Completed

18 Years

N/A

All

Phase 1;Phase 2

United States

NCT01859624
(ClinicalTrials.gov)

June 2012

8/5/2013

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement Therapy

Pompe Disease

Drug: Albuterol

Duke University

NULL

Completed

18 Years

N/A

All

Phase 1

United States