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	NCT03059160 (ClinicalTrials.gov)	April 1, 2017	2/2/2017	Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.	Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.	Rett Syndrome	Drug: Tridecanoic Acid	Sheba Medical Center	Ultragenyx Pharmaceutical Inc	Not yet recruiting	5 Years	18 Years	Female	10	Phase 2	NULL
2	NCT02696044 (ClinicalTrials.gov)	June 2016	25/2/2016	Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin	Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin: An Open-label, 10-subject Clinical Trial of UX007 (Triheptanoin) in the Treatment of Mitochondrial Dysfunction in Participants With Rett Syndrome, Dyskinesia, and Epilepsy	Rett Syndrome	Drug: triheptanoin	Center for Rare Neurological Diseases, Norcross, GA	Ultragenyx Pharmaceutical Inc;Rett Syndrome Research Trust	Recruiting	2 Years	N/A	Female	12	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

NCT03059160
(ClinicalTrials.gov)

April 1, 2017

2/2/2017

Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.

Rett Syndrome

Drug: Tridecanoic Acid

Sheba Medical Center

Ultragenyx Pharmaceutical Inc

Not yet recruiting

5 Years

18 Years

Female

Phase 2

NULL

NCT02696044
(ClinicalTrials.gov)

June 2016

25/2/2016

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin: An Open-label, 10-subject Clinical Trial of UX007 (Triheptanoin) in the Treatment of Mitochondrial Dysfunction in Participants With Rett Syndrome, Dyskinesia, and Epilepsy

Rett Syndrome

Drug: triheptanoin

Center for Rare Neurological Diseases, Norcross, GA

Ultragenyx Pharmaceutical Inc;Rett Syndrome Research Trust

Recruiting

2 Years

N/A

Female

Phase 2

United States