257. Hepatic glycogenosis Clinical trials / Disease details

Clinical trials : 14 / Drugs : 27 - (DrugBank : 7) / Drug target genes : 3 - Drug target pathways : 8

diseases per page:

Showing 1 to 10 of 14 diseases

Filter by

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	EUCTR2021-000903-19-IT (EUCTR)	14/12/2021	24/05/2021	A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)	A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics ...	Glycogen Storage Disease Type III(GSD III) MedDRA version: 20.1;Level: PT;Classification code 100532 ...	Product Name: mRNA che codifica l’enzima umano deramificante del glicogeno formulato in una nanopati ...	ULTRAGENYX PHARMACEUTICAL INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 1;Phase 2	France;United States;Canada;Spain;Germany;United Kingdom;Italy
2	EUCTR2021-000903-19-ES (EUCTR)	26/11/2021	20/07/2021	A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)	A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics ...	Glycogen Storage Disease Type III(GSD III) MedDRA version: 20.1;Level: PT;Classification code 100532 ...	Product Name: mRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle ...	Ultragenyx Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 1;Phase 2	France;United States;Canada;Spain;Germany;United Kingdom;Italy
3	NCT04990388 (ClinicalTrials.gov)	October 18, 2021	26/7/2021	Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type I ...	A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Sing ...	Glycogen Storage Disease Type III	Biological: UX053;Other: Placebo;Drug: Antipyretic;Drug: H2 Blocker;Drug: H1 Blocker	Ultragenyx Pharmaceutical Inc	NULL	Active, not recruiting	18 Years	N/A	All	8	Phase 1/Phase 2	United States;Canada;France;Italy;Spain;United Kingdom
4	NCT04930627 (ClinicalTrials.gov)	July 2021	11/6/2021	Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia	Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glyc ...	Glucose 6 Phosphatase Deficiency	Drug: Empagliflozin	Children's Memorial Health Institute, Poland	Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsa ...	Not yet recruiting	N/A	N/A	All	20	Phase 2	Poland
5	NCT03665636 (ClinicalTrials.gov)	October 16, 2020	23/8/2018	Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I	Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I	Glycogen Storage Disease Type I	Drug: Triheptanoin	Areeg El-Gharbawy	Ultragenyx Pharmaceutical Inc	Completed	1 Month	65 Years	All	4	Early Phase 1	United States
6	NCT04138251 (ClinicalTrials.gov)	June 20, 2019	20/6/2019	Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b ...	Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia ...	Glycogen Storage Disease Type I;Glucose 6 Phosphatase Deficiency	Drug: Empagliflozin	Cliniques universitaires Saint-Luc- Université Catholique de Louvain	NULL	Recruiting	1 Year	18 Years	All	5	Phase 2	Belgium
7	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 ...	Cori Forbe's Disease Also called: glycogen storage disease Type IIIor debrancher deficiency.Tarui's ...	Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive nam ...	Copenhagen Neuromuscular Center	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	Denmark
8	NCT03871673 (ClinicalTrials.gov)	April 29, 2017	9/3/2019	The Use of Uncooked Sweet Polvilho to Treat Hepatic Glycogen Storage Diseases	A Novel Approach to Treatment of Hepatic Glycogen Storage Diseases: a Study Based on the Use of Unco ...	Glycogen Storage Disease Type I	Dietary Supplement: sweet polvilho;Dietary Supplement: Cornstarch	Hospital de Clinicas de Porto Alegre	Conselho Nacional de Desenvolvimento Científico e Tecnológico;Fundação Médica do Rio Grande do Sul	Active, not recruiting	16 Years	N/A	All	11	N/A	Brazil
9	NCT03218904 (ClinicalTrials.gov)	March 17, 2017	13/7/2017	Glycogen Storage Disease Breath Test Study	Application of Novel Techniques to Devise Nutritional Therapies in Subjects With Glycogen Storage Di ...	Patient Compliance;Healthy	Dietary Supplement: Glucose intake;Dietary Supplement: Carbohydrates intake	University of British Columbia	Saudi Arabian Cultural Bureau, Ottawa;Canadian Institutes of Health Research (CIHR)	Recruiting	5 Years	35 Years	All	38	N/A	Canada
10	NCT02448667 (ClinicalTrials.gov)	January 2015	15/5/2015	Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies	Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies	Glycogen Storage Disease Type III	Dietary Supplement: FAXE Kondi;Dietary Supplement: Faxe Kondi Free	Rigshospitalet, Denmark	NULL	Recruiting	18 Years	N/A	All	15	N/A	Denmark

First Previous1 2Next Last