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	NCT01777542 (ClinicalTrials.gov)	January 2013	23/1/2013	Treatment of Rett Syndrome With Recombinant Human IGF-1	Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)	Rett Syndrome	Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1);Drug: Placebo	Boston Children's Hospital	International Rett Syndrome Foundation	Completed	2 Years	10 Years	Female	30	Phase 2	United States
2	NCT01253317 (ClinicalTrials.gov)	December 2010	2/12/2010	Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)	Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1	Rett Syndrome	Drug: rhIGF-1	Boston Children’s Hospital	International Rett Syndrome Foundation;Autism Speaks	Completed	2 Years	12 Years	Female	12	Phase 1	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

NCT01777542
(ClinicalTrials.gov)

January 2013

23/1/2013

Treatment of Rett Syndrome With Recombinant Human IGF-1

Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)

Rett Syndrome

Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1);Drug: Placebo

Boston Children's Hospital

International Rett Syndrome Foundation

Completed

2 Years

10 Years

Female

Phase 2

United States

NCT01253317
(ClinicalTrials.gov)

December 2010

2/12/2010

Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)

Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1

Rett Syndrome

Drug: rhIGF-1

Boston Children’s Hospital

International Rett Syndrome Foundation;Autism Speaks

Completed

2 Years

12 Years

Female

Phase 1

United States