DDrare: Database of Drug Development for Rare Diseases

201. アンジェルマン症候群
［臨床試験数：19，薬物数：30（DrugBank：7），標的遺伝子数：22，標的パスウェイ数：20］

Searched query = "Angelman syndrome"
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 = Aknemin; Aknemin 50; Betaine; Carbidopa; Creatine; GABOXADOL MONOHYDRATE; GTX-102; Gaboxadol; Gaboxadol monohydrate 0.5 mg; Gaboxadol monohydrate 0.5 mg (intended commercial formulation); Gaboxadol monohydrate 0.5 mg capsule; Gaboxadol monohydrate 2 mg; Gaboxadol monohydrate 2 mg (intended commercial formulation); Gaboxadol monohydrate 2 mg capsule; Gaboxadol monohydrate 5 mg; Gaboxadol monohydrate 5 mg (intended commercial formulation); Levodopa; Levodopa/Carbidopa (4:1); MINOCYCLINE; MINOCYCLINE HYDROCHLORIDE; Metafolin; Minocycline; Nutritional Formulation Containing Exogenous Ketones; OV101; OV101 Regimen 1; OV101 regimen 2; RO7248824; RO7248824/F02-01; RO7248824/F03-01 (Diluent for RO7248824/F02-01; Vitamin B12

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	NCT04259281 (ClinicalTrials.gov)	February 24, 2020	31/1/2020	A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome	A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)	Angelman Syndrome	Drug: GTX-102	GeneTX Biotherapeutics, LLC	Ultragenyx Pharmaceutical Inc	Active, not recruiting	4 Years	17 Years	All	20	Phase 1;Phase 2	United States

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

NCT04259281
(ClinicalTrials.gov)

February 24, 2020

31/1/2020

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)

Angelman Syndrome

Drug: GTX-102

GeneTX Biotherapeutics, LLC

Ultragenyx Pharmaceutical Inc

Active, not recruiting

4 Years

17 Years

All

Phase 1;Phase 2

United States