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.
| 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 | NCT03642860 (ClinicalTrials.gov) | August 15, 2018 | 20/8/2018 | The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses | Triheptanoin'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 Study | Tarui Disease;Debrancher Deficiency;GYG1 DEFICIENCY | Drug: Triheptanoin;Drug: Placebo Oil | Rigshospitalet, Denmark | Ultragenyx Pharmaceutical Inc | Unknown status | 15 Years | 85 Years | All | 20 | Phase 2 | Denmark |
| 2 | EUCTR2017-004153-17-DK (EUCTR) | 03/05/2018 | 26/03/2018 | The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses | Evaluation 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 Center | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Denmark | ||
| 3 | NCT02919631 (ClinicalTrials.gov) | October 2016 | 28/9/2016 | Triheptanoin in Mc Ardle | The Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V) | Glycogen Storage Disease Type V | Drug: Triheptanoin;Drug: Placebo oil | Institut National de la Santé Et de la Recherche Médicale, France | Rigshospitalet, Denmark;Rigshospitalet, Denmark | Not yet recruiting | 18 Years | 65 Years | Both | 12 | Phase 2 | NULL |
| 4 | NCT02432768 (ClinicalTrials.gov) | April 2015 | 4/11/2014 | The 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 V | Drug: Triheptanoin;Other: Placebo oil | Rigshospitalet, Denmark | Groupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical Inc | Completed | 18 Years | 65 Years | All | 22 | Phase 2 | Denmark |
| 5 | EUCTR2014-003644-12-DK (EUCTR) | 20/02/2015 | 04/11/2014 | The 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, Rigshospitalet | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | Denmark |