DDrare: Database of Drug Development for Rare Diseases

140. ドラベ症候群
［臨床試験数：83，薬物数：54（DrugBank：13），標的遺伝子数：48，標的パスウェイ数：62］

Searched query = "Dorabe syndrome", "Dravet 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 = Ataluren; CANNABIDIOL; CBD; CBD - Oral Solution, is known as Epidiolex, and is the approved name in the USA.; Cannabidiol; Cannabidiol (CBD); Cannabidiol Oral Solution; Carbidopa; Clemizole; Clobazam; DIACOMIT; DIACOMIT (Stiripentol); Diacomit; EPX-100 (Clemizole HCl); EPX-100 (Clemizole Hydrochloride); FENFLURAMINE HYDROCHLORIDE; FENFLURAMINE HYDROCHLORIDE (colorless); FENFLURAMINE HYDROCHLORIDE (red); FRISIUM*30CPS 10MG; Fenfluramina; Fenfluramina Cloridrato; Fenfluramine; Fenfluramine Hydrochloride; Fenfluramine hydrochloride; Fenfluramine hydrochloride (colorless); Fenfluramine hydrochloride (red); Frisium; GWP42003-P; GWP42003-P 10 mg/kg/day Dose; GWP42003-P 20 mg/kg/day Dose; GWP42003-P 5 mg/kg/day Dose; Levodopa; Lorcaserin; ME2080 (stiripentol); None; None at this time; Other antiepileptics; Placebo; Procedure: 12-Lead ECG; STK-001; Stiripentol; Stiripentolo; TAK-935; TOPAMAX; Topiramate; Turmeric; Verapamil; ZX008; ZX008 (Fenfluramine Hydrochloride); ZX008 - 0.2 mg/kg/day; ZX008 - 0.4 mg/kg/day; ZX008 - 0.8 mg/kg/day; ZX008 - 20 mg/day maximum dose; ZX008 0.2 and 0.8 mg/kg/day

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	NCT02758626 (ClinicalTrials.gov)	November 2016	4/4/2016	Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome	A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome	Epilepsy	Drug: ataluren;Drug: Placebo	NYU Langone Health	PTC Therapeutics	Active, not recruiting	2 Years	12 Years	All	16	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

NCT02758626
(ClinicalTrials.gov)

November 2016

4/4/2016

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome

Epilepsy

Drug: ataluren;Drug: Placebo

NYU Langone Health

PTC Therapeutics

Active, not recruiting

2 Years

12 Years

All

Phase 2

United States