DDrare: Database of Drug Development for Rare Diseases

201. アンジェルマン症候群　［臨床試験数：12，薬物数：15（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 of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Drug = Aknemin 50; Betaine; Carbidopa; Creatine; Gaboxadol; Levodopa; Levodopa/carbidopa (4:1); Metafolin; Minocycline; Minocycline hydrochloride; Nutritional formulation containing exogenous ketones; Ov101; Ov101 regimen 1; Ov101 regimen 2; Vitamin b12

No.	TrialID	Date_ enrollement	Last_Refreshed_ on	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04106557	September 9, 2019	14 October 2019	A Study of OV101 in Individuals With Angelman Syndrome (AS)	A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome	Primary Disease or Condition Being Studied: Angelman Syndrome (AS)	Drug: Gaboxadol;Drug: Placebo	Ovid Therapeutics Inc.		Recruiting	2 Years	12 Years	All	65	Phase 3	United States
2	NCT03882918	February 25, 2019	8 April 2019	An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome	An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome	Angelman Syndrome	Drug: OV101	Ovid Therapeutics Inc.		Recruiting	13 Years	49 Years	All	82	Phase 2	United States
3	NCT03644693	August 13, 2018	3 September 2018	Nutritional Formulation for Angelman Syndrome	Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome	Angelman Syndrome	Dietary Supplement: Nutritional Formulation Containing Exogenous Ketones;Dietary Supplement: Placebo Formulation	Vanderbilt University Medical Center		Recruiting	4 Years	11 Years	All	15	N/A	United States
4	NCT03109756	April 3, 2017	16 December 2017	Single Dose Pharmacokinetic (PK) Study	A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome	Angelman Syndrome;Fragile X Syndrome	Drug: OV101	Ovid Therapeutics Inc.		Not recruiting	13 Years	17 Years	All	12	Phase 1	United States
5	NCT02996305	January 2016	20 August 2018	A Study in Adults and Adolescents With Angelman Syndrome	A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol	Angelman Syndrome	Drug: OV101 Regimen 1;Drug: OV101 regimen 2;Other: Placebo	Ovid Therapeutics Inc.		Not recruiting	13 Years	49 Years	All	88	Phase 2	United States;Israel
No.	TrialID	Date_ enrollement	Last_Refreshed_ on	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	NCT02056665	January 2014	2 November 2015	Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome	Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome	Angelman Syndrome	Drug: MINOCYCLINE;Drug: PLACEBO (for Minocycline)	Puerta de Hierro University Hospital		Not recruiting	6 Years	30 Years	Both	32	Phase 2	Spain
7	EUCTR2013-002154-67-ES	26/12/2013	27 January 2014	STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)	RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY) - A-MANECE	Angelman Syndrome MedDRA version: 16.0 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]	Trade Name: Aknemin 50 Pharmaceutical Form: Capsule INN or Proposed INN: MINOCYCLINE CAS Number: 13614-98-7 Current Sponsor code: PL 33016/0005 Other descriptive name: MINOCYCLINE HYDROCHLORIDE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Capsule Route of administration of the placebo: Oral use	Dra. Cristina Avendaño Solá		Authorised			Female: yes Male: yes			Spain
8	NCT03235037	November 26, 2013	29 April 2019	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		Not recruiting	18 Months	16 Years	All	10	N/A
9	NCT01531582	April 2012	19 February 2015	Minocycline in the Treatment of Angelman Syndrome	The Efficacy of Minocycline in the Treatment of Angelman Syndrome	Angelman Syndrome	Drug: minocycline	University of South Florida		Not recruiting	4 Years	12 Years	Both	25	N/A	United States
10	NCT01281475	January 2011	19 October 2017	A Trial of Levodopa in Angelman Syndrome	A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome	Angelman Syndrome	Drug: Placebo Oral Capsule;Drug: Levodopa	Wen-Hann Tan	Rady Children's Hospital, San Diego;University of California, San Francisco;Baylor College of Medicine;Vanderbilt University;Greenwood Genetic Center;Children's Hospital Medical Center, Cincinnati;Angelman Syndrome Foundation, Inc.	Not recruiting	4 Years	12 Years	All	67	Phase 2/Phase 3	United States
No.	TrialID	Date_ enrollement	Last_Refreshed_ on	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
11	NCT00829439	January 2009	19 October 2017	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		Not recruiting	4 Years	12 Years	All	16	Phase 1	United States
12	NCT00348933	July 2006	19 October 2017	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	Not recruiting	N/A	5 Years	All	90	N/A	United States

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