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	NCT02367014 (ClinicalTrials.gov)	February 2015	9/2/2015	Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy	Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial Disease	Mitochondrial Myopathy	Drug: elamipretide (low dose);Drug: elamipretide (intermediate dose);Drug: elamipretide (high dose);Drug: Placebo	Stealth BioTherapeutics Inc.	NULL	Completed	16 Years	65 Years	All	36	Phase 1;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

NCT02367014
(ClinicalTrials.gov)

February 2015

9/2/2015

Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial Disease

Mitochondrial Myopathy

Drug: elamipretide (low dose);Drug: elamipretide (intermediate dose);Drug: elamipretide (high dose);Drug: Placebo

Stealth BioTherapeutics Inc.

NULL

Completed

16 Years

65 Years

All

Phase 1;Phase 2

United States