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	NCT02715115 (ClinicalTrials.gov)	March 2016	21/2/2016	A Safety Study of NNZ-2566 in Pediatric Rett Syndrome	A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome	Rett Syndrome	Drug: NNZ-2566;Drug: Placebo	Neuren Pharmaceuticals Limited	rettsyndrome.org	Completed	5 Years	15 Years	Female	82	Phase 2	United States
2	NCT01703533 (ClinicalTrials.gov)	March 2013	4/10/2012	A Safety Study of NNZ-2566 in Patients With Rett Syndrome	A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett Syndrome	Rett Syndrome	Drug: NNZ-2566;Drug: Placebo	Neuren Pharmaceuticals Limited	Baylor College of Medicine;International Rett Syndrome Foundation	Completed	16 Years	45 Years	Female	67	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

NCT02715115
(ClinicalTrials.gov)

March 2016

21/2/2016

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome

Rett Syndrome

Drug: NNZ-2566;Drug: Placebo

Neuren Pharmaceuticals Limited

rettsyndrome.org

Completed

5 Years

15 Years

Female

Phase 2

United States

NCT01703533
(ClinicalTrials.gov)

March 2013

4/10/2012

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett Syndrome