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 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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03642860August 15, 201820/8/2018The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With GlycogenosesTriheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over StudyTarui Disease;Debrancher Deficiency;GYG1 DEFICIENCYDrug: Triheptanoin;Drug: Placebo OilRigshospitalet, DenmarkUltragenyx Pharmaceutical IncUnknown status15 Years85 YearsAll20Phase 2Denmark
2EUCTR2017-004153-17-DK03/05/201826/03/2018The effect of Triheptanoin on excercise in adults and adolescence with glycogenosesEvaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV.
MedDRA version: 20.1Level: PTClassification code 10053241Term: Glycogen storage disease type VIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: PTClassification code 10053250Term: Glycogen storage disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10053255Term: Tarui diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10016983Term: Forbes' diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Copenhagen Neuromuscular CenterNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Denmark
3NCT02919631October 201628/9/2016Triheptanoin in Mc ArdleThe Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Triheptanoin;Drug: Placebo oilInstitut National de la Santé Et de la Recherche Médicale, FranceRigshospitalet, Denmark;Rigshospitalet, DenmarkNot yet recruiting18 Years65 YearsBoth12Phase 2NULL
4NCT02432768April 20154/11/2014The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Triheptanoin;Other: Placebo oilRigshospitalet, DenmarkGroupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical IncCompleted18 Years65 YearsAll22Phase 2Denmark
5EUCTR2014-003644-12-DK20/02/201504/11/2014The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V)The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V) - Triheptanoin in McArdle McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency
MedDRA version: 19.0Level: LLTClassification code 10026970Term: McArdles diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0Level: LLTClassification code 10026969Term: McArdle's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0Level: PTClassification code 10018462Term: Glycogen storage disease type VSystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Neuromuscular Research Unit, RigshospitaletNULLNot RecruitingFemale: yes
Male: yes
28Phase 2Denmark