EPI-743 ( DrugBank: - )

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3 diseases

ID	Disease name (Link within this page)	Number of trials
21	Mitochondrial disease	1
156	Rett syndrome	2
337	Homocystinuria	1

21. Mitochondrial disease

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Clinical trials : 39 /  Drugs : 42 - (DrugBank : 32) / Drug target genes : 47 - Drug target pathways : 67

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	NCT01642056 (ClinicalTrials.gov)	September 1, 2012	14/7/2012	EPI-743 for Metabolism or Mitochondrial Disorders	Therapeutic Trial of EPI -743 In Patients With Disorders of Energy Utilization or Oxidation-Reduction	Mitochondrial Disease;Neurology;Myoptahy	Drug: EPI-743;Drug: Placebo	National Human Genome Research Institute (NHGRI)	NULL	Completed	2 Years	11 Years	All	20	Phase 1/Phase 2	United States

156. Rett syndrome

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Clinical trials : 44 /  Drugs : 61 - (DrugBank : 23) / Drug target genes : 57 - Drug target pathways : 83

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	EUCTR2012-005021-76-IT (EUCTR)	31/01/2013	11/12/2012	Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.	A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome	Rett Syndrome MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: EPI-743 Product Code: NA Other descriptive name: Alpha-tocotrienol quinone	EDISON PHARMACEUTICALS INC.	NULL	Not Recruiting			Female: yes Male: no		Phase 2	Italy
2	NCT01822249 (ClinicalTrials.gov)	January 2013	25/3/2013	Phase 2 Study of EPI-743 for Treatment of Rett Syndrome	A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome	Rett Syndrome	Drug: EPI-743;Drug: Placebo	Edison Pharmaceuticals Inc	NULL	Completed	N/A	18 Years	Female	24	Phase 2	Italy

337. Homocystinuria

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Clinical trials : 16 /  Drugs : 19 - (DrugBank : 7) / Drug target genes : 2 - Drug target pathways : 23

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	NCT01793090 (ClinicalTrials.gov)	January 2013	8/2/2013	EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment	Phase 2, Double-Blind, Placebo Controlled Clinical Trial of EPI-743 in Subjects With Cobalamin C Defect	Methylmalonic Aciduria and Homocystinuria,Cblc Type;Genetic Disease;Retinopathy	Drug: Epi-743;Other: Placebo supplementation	Bambino Gesù Hospital and Research Institute	Catholic University of the Sacred Heart	Completed	1 Year	20 Years	All	30	Phase 2	Italy