DDrare: Database of Drug Development for Rare Diseases

156. レット症候群
［臨床試験数：36，薬物数：49（DrugBank：19），標的遺伝子数：77，標的パスウェイ数：112］

Searched query = "Rett 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 = ANA001XHCl (Syntagon) or VEXA-04 (Patheon); ANAVEX2-73; ANAVEX2-73 (blarcamesine); ANAVEX2-73 oral liquid; Acetate; Administration of a high dose of desipramine; Administration of a low dose of desipramine; Alpha-tocotrienol quinone; Blarcamesine; CANNABIDIOL; CBD; CBD - Oral Solution, is known as Epidyolex, and is the approved name; CBD Oral solution, is known as Epidyolex and is the approved name in the EU; Cannabidiol; Cannabidiol (CBD); Creatine; Creatine monohydrate; Desipramine; Dextromethorphan; Donepezil; Donepezil hydrochloride; EMD 128130; EPI-743; Epidyolex; Epidyolex and is the approved name in the EU; Fingolimod; Fingolimod (FTY720); GWP42003-P; Glatiramer; Glatiramer Acetate; Glatiramer Acetate (Copaxone®); Human insulin; Ketamine; Lactobacillus plantarum; Lovastatin; NNZ-2566; Probiotic-Lactobacillus plantarum PS128; Recombinant Human Insulin Growth Factor 1 (rhIGF-1); Rett-T; RhIGF-1; Risperidone; SARIZOTAN HYDROCHLORIDE; Sarizotan; Sarizotan hydrochloride; Tocotrienol; Topiramate; Tridecanoic Acid; Triheptanoin; Trofinetide

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	NCT04304482 (ClinicalTrials.gov)	July 1, 2020	8/3/2020	ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome	A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome	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	18 Years	Female	69	Phase 2;Phase 3	Australia
2	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 with Rett Syndrome - The Safety and Efficacy of ANAVEX2-73 in Patients with Rett Syndrome	Rett Syndrome MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: ANAVEX2-73 INN or Proposed INN: ANAVEX2-73 (blarcamesine) Other descriptive name: ANA001XHCl (Syntagon) or VEXA-04 (Patheon)	Anavex Germany GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: no	30	Phase 2	Australia;United Kingdom
3	NCT03941444 (ClinicalTrials.gov)	May 6, 2019	6/5/2019	ANAVEX2-73 Study in Patients With Rett Syndrome	A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome	Rett Syndrome	Drug: ANAVEX2-73;Drug: Placebos	Anavex Life Sciences Corp.	NULL	Recruiting	18 Years	45 Years	Female	30	Phase 2	Australia;United Kingdom
4	NCT03758924 (ClinicalTrials.gov)	February 28, 2019	25/11/2018	Study of ANAVEX2-73 in Patients With Rett Syndrome	A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome	Rett Syndrome	Drug: ANAVEX2-73;Drug: Placebo	Anavex Life Sciences Corp.	International Rett Syndrome Foundation Rettsyndrome.org	Active, not recruiting	18 Years	45 Years	Female	31	Phase 2	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

NCT04304482
(ClinicalTrials.gov)

July 1, 2020

8/3/2020

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome

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

18 Years

Female

Phase 2;Phase 3

Australia

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 with Rett Syndrome - The Safety and Efficacy of ANAVEX2-73 in Patients with Rett Syndrome

Rett Syndrome
MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: ANAVEX2-73
INN or Proposed INN: ANAVEX2-73 (blarcamesine)
Other descriptive name: ANA001XHCl (Syntagon) or VEXA-04 (Patheon)

Anavex Germany GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: no

Phase 2

Australia;United Kingdom

NCT03941444
(ClinicalTrials.gov)

May 6, 2019

6/5/2019

ANAVEX2-73 Study in Patients With Rett Syndrome

A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome

Rett Syndrome

Drug: ANAVEX2-73;Drug: Placebos

Anavex Life Sciences Corp.

NULL

Recruiting

18 Years

45 Years

Female

Phase 2

Australia;United Kingdom

NCT03758924
(ClinicalTrials.gov)

February 28, 2019

25/11/2018

Study of ANAVEX2-73 in Patients With Rett Syndrome

A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome

Rett Syndrome

Drug: ANAVEX2-73;Drug: Placebo

Anavex Life Sciences Corp.

International Rett Syndrome Foundation Rettsyndrome.org

Active, not recruiting

18 Years

45 Years

Female

Phase 2

United States