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	EUCTR2012-005021-76-IT (EUCTR)	31/01/2013	11/12/2012	Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.	A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome	Rett Syndrome MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: EPI-743 Product Code: NA Other descriptive name: Alpha-tocotrienol quinone	EDISON PHARMACEUTICALS INC.	NULL	Not Recruiting			Female: yes Male: no		Phase 2A	Italy

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EUCTR2012-005021-76-IT
(EUCTR)

31/01/2013

11/12/2012

Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.

A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome

Rett Syndrome
MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: EPI-743
Product Code: NA
Other descriptive name: Alpha-tocotrienol quinone

EDISON PHARMACEUTICALS INC.

NULL

Not Recruiting

Female: yes
Male: no

Phase 2A

Italy