DDrare: Database of Drug Development for Rare Diseases

Vatiquinone (DrugBank: Vatiquinone)

2 diseases

ID	Disease name (Link within this page)	Number of trials
18	Spinocerebellar degeneration	1
21	Mitochondrial disease	1

18. Spinocerebellar degeneration [ 59 clinical trials, 78 drugs, (DrugBank: 28 drugs), 44 drug target genes, 59 drug target pathways]

Searched query = "Spinocerebellar degeneration", "SCD", "Spinocerebellar ataxia type I", "SCA1", "Spinocerebellar ataxia type II", "SCA2", "Spinocerebellar ataxia type III", "SCA3", "Machado-Joseph disease", "Spinocerebellar ataxia type VI", "SCA6", "Spinocerebellar ataxia type VII", "SCA7", "Spinocerebellar ataxia type X", "SCA10", "Spinocerebellar ataxia type XII", "SCA12", "Dentatorubural pallidoluysian atrophy", "Dentatorubropallidoluysial atrophy", "DRPLA", "Naito-Koyanagi disease", "Friedreich ataxia", "FRDA", "Ataxia with vitamin E deficiency", "AVED", "Early-onset ataxia with ocular motor ataxia and hypoalbuminemia", "EOAH"
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.

1 / 59 trial found

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	NCT04577352 (ClinicalTrials.gov)	November 17, 2020	30/9/2020	A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)	Friedreich Ataxia	Drug: Vatiquinone;Drug: Placebo	PTC Therapeutics	NULL	Not yet recruiting	7 Years	N/A	All	126	Phase 2;Phase 3	United States;Australia;Canada;Italy

21. Mitochondrial disease [ 23 clinical trials, 29 drugs, (DrugBank: 28 drugs), 44 drug target genes, 65 drug target pathways]

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.

1 / 23 trial found

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