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	EUCTR2019-004478-24-DE (EUCTR)	05/06/2020	26/02/2020	An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)	An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA) - ELARA	Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Gaboxadol monohydrate 0.5 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 2 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE Product Name: Gaboxadol monohydrate 5 mg (intended commercial formulation) Product Code: OV101 INN or Proposed INN: Gaboxadol Other descriptive name: GABOXADOL MONOHYDRATE	Ovid Therapeutics Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	170	Phase 3	United States;Australia;Israel;Netherlands;Germany

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

EUCTR2019-004478-24-DE
(EUCTR)

05/06/2020

26/02/2020

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA) - ELARA

Angelman Syndrome
MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Gaboxadol monohydrate 0.5 mg (intended commercial formulation)
Product Code: OV101
INN or Proposed INN: Gaboxadol
Other descriptive name: GABOXADOL MONOHYDRATE
Product Name: Gaboxadol monohydrate 2 mg (intended commercial formulation)
Product Code: OV101
INN or Proposed INN: Gaboxadol
Other descriptive name: GABOXADOL MONOHYDRATE
Product Name: Gaboxadol monohydrate 5 mg (intended commercial formulation)
Product Code: OV101
INN or Proposed INN: Gaboxadol
Other descriptive name: GABOXADOL MONOHYDRATE

Ovid Therapeutics Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

170

Phase 3

United States;Australia;Israel;Netherlands;Germany