256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105

  
5 trials found
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1NCT03642860
(ClinicalTrials.gov)
August 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 IncTerminated15 Years85 YearsAll3Phase 2Denmark
2EUCTR2017-004153-17-DK
(EUCTR)
03/05/201826/03/2018The 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: 20.1;Level: PT;Classification code 10053241;Term: Glycogen storage disease type VII;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10053255;Term: Tarui disease;Classification code 10016983;Term: Forbes' disease;System 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
3NCT02919631
(ClinicalTrials.gov)
October 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
4NCT02432768
(ClinicalTrials.gov)
April 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-DK
(EUCTR)
20/02/201504/11/2014The 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: 19.0;Level: LLT;Classification code 10026970;Term: McArdles disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0;Classification code 10026969;Term: McArdle's disease;Level: PT;Classification code 10018462;Term: Glycogen storage disease type V;System 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