21. Mitochondrial disease Clinical trials / Disease details
Clinical trials : 33 / 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 | EUCTR2018-001094-25-IT (EUCTR) | 12/09/2019 | 20/05/2021 | Clinical trial with Phenilbutirrate to reduce lactic acid in patients affected with Melas Syndrome and PHD deficency encephalopathy | Phenylbutyrate Therapy in Mitochondrial Disease with lactic acidosis: an opel label clinical trial in MELAS and PHD deficiency patients - PHEMI | Melas Syndrome and PDH deficency enchephalopathy MedDRA version: 20.0;Level: PT;Classification code 10053872;Term: MELAS syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10062950;Term: Leigh syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Feburane Product Name: pheburane Product Code: [A042917017] | FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 2 | Italy |