156. Rett syndrome Clinical trials / Disease details

Clinical trials : 40 / Drugs : 53 - (DrugBank : 19) / Drug target genes : 77 - Drug target pathways : 113

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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	NCT04988867 (ClinicalTrials.gov)	September 22, 2021	23/7/2021	An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett ...	An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett ...	Rett Syndrome	Drug: Trofinetide	ACADIA Pharmaceuticals Inc.	NULL	Recruiting	2 Years	5 Years	Female	10	Phase 2/Phase 3	United States
2	NCT04776746 (ClinicalTrials.gov)	November 8, 2020	29/11/2020	Open-Label Extension Study of Trofinetide for Rett Syndrome	An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome	Rett Syndrome	Drug: trofinetide	ACADIA Pharmaceuticals Inc.	NULL	Enrolling by invitation	5 Years	22 Years	Female	153	Phase 3	United States
3	EUCTR2019-001605-24-IT (EUCTR)	02/11/2020	21/01/2021	An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome	An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP4 ...	Rett syndrome(RTT) [typical or atypical] MedDRA version: 20.0;Level: PT;Classification code 10077709 ...	Trade Name: CBD soluzione orale nota come Epidyolex e con tale nome è stato approvato in UE Product Na ...	GW RESEARCH LTD.	NULL	NA			Female: yes Male: no	252	Phase 3	United States;Canada;Spain;Australia;United Kingdom;Italy
4	NCT04304482 (ClinicalTrials.gov)	July 1, 2020	8/3/2020	ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome	ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy S ...	Rett Syndrome	Drug: ANAVEX2-73 oral liquid;Drug: Placebo oral liquid	Anavex Life Sciences Corp.	Anavex Australia Pty Ltd.;Anavex Germany GmbH	Recruiting	5 Years	17 Years	Female	84	Phase 2/Phase 3	Australia;Canada
5	EUCTR2019-004345-32-GB (EUCTR)	26/05/2020	07/02/2020	The use of ANAVEX2-73 as a new therapeutic treatment for Patients with Rett Syndrome	A Double-Blind, Randomised, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients ...	Rett Syndrome MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System ...	Product Name: ANAVEX2-73 INN or Proposed INN: ANAVEX2-73 (blarcamesine) Other descriptive name: ANA001X ...	Anavex Germany GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: no	30	Phase 2	Australia;United Kingdom
6	EUCTR2019-001605-24-ES (EUCTR)	04/03/2020	21/02/2020	An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome	An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP4 ...	Rett syndrome(RTT) [typical or atypical] MedDRA version: 20.0;Level: PT;Classification code 10077709 ...	Trade Name: Epidyolex and is the approved name in the EU Product Name: Cannabidiol (CBD) Product Code: G ...	GW Research Ltd	NULL	Not Recruiting			Female: yes Male: no	252	Phase 3	United States;Canada;Spain;Australia;United Kingdom;Italy
7	NCT04252586 (ClinicalTrials.gov)	February 28, 2020	30/1/2020	An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Ret ...	An Open-label Extension Trial to Investigate the Long-term Safety of Cannabidiol Oral Solution (GWP4 ...	Rett Syndrome;RTT	Drug: GWP42003-P	GW Research Ltd	NULL	Terminated	2 Years	18 Years	All	21	Phase 3	United States;Australia;Canada;Italy;Spain;United Kingdom
8	NCT04279314 (ClinicalTrials.gov)	January 29, 2020	19/2/2020	Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome	A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett ...	Rett Syndrome	Drug: Trofinetide	ACADIA Pharmaceuticals Inc.	NULL	Active, not recruiting	5 Years	21 Years	Female	180	Phase 3	United States
9	EUCTR2019-001605-24-GB (EUCTR)	15/01/2020	23/10/2019	An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome	An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP4 ...	Rett syndrome(RTT) [typical or atypical] MedDRA version: 20.0;Level: PT;Classification code 10077709 ...	Trade Name: CBD Oral solution, is known as Epidyolex and is the approved name in the EU Product Name: ...	GW Research Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	252	Phase 3	United States;France;Canada;Spain;Australia;Italy;United Kingdom
10	NCT04181723 (ClinicalTrials.gov)	November 6, 2019	26/11/2019	Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)	A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatmen ...	Rett Syndrome	Drug: Trofinetide;Other: Placebo	ACADIA Pharmaceuticals Inc.	NULL	Completed	5 Years	20 Years	Female	187	Phase 3	United States

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