DDrare: Database of Drug Development for Rare Diseases

257. 肝型糖原病
［臨床試験数：10，薬物数：19（DrugBank：6），標的遺伝子数：1，標的パスウェイ数：6］

Searched query = "Hepatic glycogenosis", "Liver glycogenosis", "Hepatic glycogen storage disease", "Liver glycogen storage disease", "Glycogen storage disease type I", "GSDI", "von Gierke disease", "Glucose-6-phosphatase deficiency", "G6Pase 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", "Adult polyglucosan body disease", "Glycogen storage disease type VI", "GSDVI", "Hers disease", "Hepatic phosphorylase deficiency", "Liver phosphorylase deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase 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.

Drug = Carbohydrates intake; Cornstarch; EPHYNAL; Empagliflozin; FAXE Kondi; Faxe Kondi Free; Glucose; Glucose intake; Glycosade; Other: Vegetable Oil; QUARK; Quark; Ramipril; Sweet polvilho; TRIHEPTANOIN; Tocopherol; Tocopherol (vit E); Triheptanoin; UX007

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	EUCTR2006-005449-12-IT (EUCTR)	11/10/2006	23/05/2007	EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b - GSD1: study of specific therapeutic intervention	EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b - GSD1: study of specific therapeutic intervention	As concern objective number 1: Patients affected by glycogen storage disease (GSD) type 1a and patients affected by GSD1b with renal dysfunction including glomerular hyperfiltration, microalbuminuria and/or proteinuria As concern objective number 2: Patients affected by GSD1b showing neutropenia MedDRA version: 9.1;Level: LLT;Classification code 10018464;Term: Glycogen storage disease type I	Trade Name: QUARK INN or Proposed INN: Ramipril Trade Name: EPHYNAL INN or Proposed INN: Tocopherol (vit E)	Dipartimento di Pediatria	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Italy

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

EUCTR2006-005449-12-IT
(EUCTR)

11/10/2006

23/05/2007

EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b - GSD1: study of specific therapeutic intervention

As concern objective number 1: Patients affected by glycogen storage disease (GSD) type 1a and patients affected by GSD1b with renal dysfunction including glomerular hyperfiltration, microalbuminuria and/or proteinuria As concern objective number 2: Patients affected by GSD1b showing neutropenia
MedDRA version: 9.1;Level: LLT;Classification code 10018464;Term: Glycogen storage disease type I

Trade Name: QUARK
INN or Proposed INN: Ramipril
Trade Name: EPHYNAL
INN or Proposed INN: Tocopherol (vit E)

Dipartimento di Pediatria

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Italy