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	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 (GWP42003-P, CBD-OS) in patients with Rett Syndrome	Rett syndrome (RTT) [typical or atypical] 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]	Trade Name: CBD Oral solution, is known as Epidyolex and is the approved name in the EU Product Name: Cannabidiol (CBD) Product Code: GWP42003-P INN or Proposed INN: Cannabidiol Other descriptive name: CANNABIDIOL	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
2	EUCTR2018-003370-27-GB (EUCTR)	09/04/2019	24/12/2018	A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.	A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.	Rett syndrome (RTT) [typical or atypical] 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]	Trade Name: CBD - Oral Solution, is known as Epidyolex, and is the approved name Product Name: Cannabidiol (CBD) Product Code: GWP42003-P INN or Proposed INN: Cannabidiol Other descriptive name: CANNABIDIOL	GW Research Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	252	Phase 2;Phase 3	United States;France;Canada;Spain;Australia;Italy;United Kingdom

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

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 (GWP42003-P, CBD-OS) in patients with Rett Syndrome

Rett syndrome (RTT) [typical or atypical]
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]

Trade Name: CBD Oral solution, is known as Epidyolex and is the approved name in the EU
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
INN or Proposed INN: Cannabidiol
Other descriptive name: CANNABIDIOL

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

EUCTR2018-003370-27-GB
(EUCTR)

09/04/2019

24/12/2018

A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.

Trade Name: CBD - Oral Solution, is known as Epidyolex, and is the approved name
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
INN or Proposed INN: Cannabidiol
Other descriptive name: CANNABIDIOL

GW Research Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

252

Phase 2;Phase 3

United States;France;Canada;Spain;Australia;Italy;United Kingdom