DDrare: Database of Drug Development for Rare Diseases

21. ミトコンドリア病
［臨床試験数：23，薬物数：29（DrugBank：28），標的遺伝子数：44，標的パスウェイ数：65］

Searched query = "Mitochondrial disease"
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 = Bupropion; Caffeine; CoenzymeQ10; Cysteamine; Cysteamine Bitartrate; Dexmedetomidine; Dextromethorphan; EPI-743; Elamipretide; Elamipretide (high dose); Elamipretide (intermediate dose); Elamipretide (low dose); Flurbiprofen; Fluzone; Fluzone®; KH176; KL1333; Low Residue Diet Intervention; Midazolam; Midazolam injection; Omeprazole; Propofol; RG2133 (2',3',5'-tri-O-acetyluridine); Repaglinide; SPP-004; Sevoflurane; Sonlicromanol; Tri-O-acetyluridine; Vatiquinone

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	NCT04378075 (ClinicalTrials.gov)	September 28, 2020	4/5/2020	A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy	Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy	Mitochondrial Diseases;Drug Resistant Epilepsy;Leigh Disease;Leigh Syndrome;Mitochondrial Encephalopathy (MELAS);Pontocerebellar Hypoplasia Type 6 (PCH6);Alpers Disease;Alpers Syndrome	Drug: Vatiquinone;Other: Placebo	PTC Therapeutics	NULL	Recruiting	N/A	18 Years	All	60	Phase 2;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

NCT04378075
(ClinicalTrials.gov)

September 28, 2020

4/5/2020

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy

Mitochondrial Diseases;Drug Resistant Epilepsy;Leigh Disease;Leigh Syndrome;Mitochondrial Encephalopathy (MELAS);Pontocerebellar Hypoplasia Type 6 (PCH6);Alpers Disease;Alpers Syndrome

Drug: Vatiquinone;Other: Placebo

PTC Therapeutics

NULL

Recruiting

N/A

18 Years

All

Phase 2;Phase 3

United States