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	NCT03235037 (ClinicalTrials.gov)	November 26, 2013	28/7/2017	Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome	Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome	Angelman Syndrome	Drug: Levodopa	Bennett Lavenstein	NULL	Completed	18 Months	16 Years	All	10	N/A	NULL
2	NCT01281475 (ClinicalTrials.gov)	January 2011	20/1/2011	A Trial of Levodopa in Angelman Syndrome	A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome	Angelman Syndrome	Drug: Levodopa;Drug: Placebo Oral Capsule	Wen-Hann Tan	Rady Children's Hospital, San Diego;University of California, San Francisco;Baylor College of Medicine;Vanderbilt University Medical Center;Greenwood Genetic Center;Children's Hospital Medical Center, Cincinnati;Angelman Syndrome Foundation, Inc.	Completed	4 Years	12 Years	All	67	Phase 2;Phase 3	United States
3	NCT00829439 (ClinicalTrials.gov)	January 2009	26/1/2009	Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome	A Dose-escalation Tolerability Study of Levodopa/Carbidopa in Angelman Syndrome	Angelman Syndrome	Drug: Levodopa /Carbidopa (4:1)	Boston Children’s Hospital	NULL	Completed	4 Years	12 Years	All	16	Phase 1	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

NCT03235037
(ClinicalTrials.gov)

November 26, 2013

28/7/2017

Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome

Angelman Syndrome

Drug: Levodopa

Bennett Lavenstein

NULL

Completed

18 Months

16 Years

All

N/A

NULL

NCT01281475
(ClinicalTrials.gov)

January 2011

20/1/2011

A Trial of Levodopa in Angelman Syndrome

A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome

Angelman Syndrome

Drug: Levodopa;Drug: Placebo Oral Capsule

Wen-Hann Tan

Rady Children's Hospital, San Diego;University of California, San Francisco;Baylor College of Medicine;Vanderbilt University Medical Center;Greenwood Genetic Center;Children's Hospital Medical Center, Cincinnati;Angelman Syndrome Foundation, Inc.

Completed

4 Years

12 Years

All

Phase 2;Phase 3

United States

NCT00829439
(ClinicalTrials.gov)

January 2009

26/1/2009

Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome

A Dose-escalation Tolerability Study of Levodopa/Carbidopa in Angelman Syndrome

Angelman Syndrome

Drug: Levodopa /Carbidopa (4:1)

Boston Children’s Hospital

NULL

Completed

4 Years

12 Years

All

Phase 1

United States