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	EUCTR2020-003124-16-NL (EUCTR)		20/10/2020	The KHENERGYC study: a placebo controlled, double-blind study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with a mitochondrial disease.	A randomized placebo controlled, double-blind phase II study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease.	Genetically confirmed mitochondrial disease;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: Sonlicromanol Product Code: KH176 INN or Proposed INN: sonlicromanol Other descriptive name: KH176	Khondrion B.V.	NULL	NA			Female: yes Male: yes	24	Phase 2	Netherlands

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EUCTR2020-003124-16-NL
(EUCTR)

20/10/2020

The KHENERGYC study: a placebo controlled, double-blind study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with a mitochondrial disease.

A randomized placebo controlled, double-blind phase II study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease.

Genetically confirmed mitochondrial disease;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: Sonlicromanol
Product Code: KH176
INN or Proposed INN: sonlicromanol
Other descriptive name: KH176

Khondrion B.V.

NULL

Female: yes
Male: yes

Phase 2

Netherlands