DDrare: Database of Drug Development for Rare Diseases

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	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 Syndrome	Rett Syndrome	Drug: Trofinetide	ACADIA Pharmaceuticals Inc.	NULL	Enrolling by invitation	5 Years	21 Years	Female	180	Phase 3	United States
2	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 Treatment of Girls and Women With Rett Syndrome	Rett Syndrome	Drug: Trofinetide;Other: Placebo	ACADIA Pharmaceuticals Inc.	NULL	Recruiting	5 Years	20 Years	Female	184	Phase 3	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

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 Syndrome

Rett Syndrome

Drug: Trofinetide

ACADIA Pharmaceuticals Inc.

NULL

Enrolling by invitation

5 Years

21 Years

Female

180

Phase 3

United States

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 Treatment of Girls and Women With Rett Syndrome