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	NCT00348933 (ClinicalTrials.gov)	July 2006	3/7/2006	Dietary Supplements for the Treatment of Angelman Syndrome	Efficacy of a Therapeutic Treatment Trial in Angelman Syndrome	Angelman Syndrome;Nervous System Diseases	Drug: Betaine;Drug: Creatine;Drug: Metafolin;Drug: Vitamin B12	University of California, San Diego	Baylor College of Medicine;Rady Children's Hospital, San Diego;Boston Children’s Hospital;Greenwood Genetic Center;Rare Diseases Clinical Research Network	Completed	N/A	5 Years	All	90	N/A	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

NCT00348933
(ClinicalTrials.gov)

July 2006

3/7/2006

Dietary Supplements for the Treatment of Angelman Syndrome

Efficacy of a Therapeutic Treatment Trial in Angelman Syndrome

Angelman Syndrome;Nervous System Diseases

Drug: Betaine;Drug: Creatine;Drug: Metafolin;Drug: Vitamin B12

University of California, San Diego

Baylor College of Medicine;Rady Children's Hospital, San Diego;Boston Children’s Hospital;Greenwood Genetic Center;Rare Diseases Clinical Research Network

Completed

N/A

5 Years

All

N/A

United States