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.
| 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 | EUCTR2016-001696-79-NL (EUCTR) | 06/09/2016 | 18/08/2016 | A Phase II study with KH176 in patients with mitochondrial disease | A double-blind, randomized, placebo-controlled, single-center, two-way cross-over study with KH176 in patients with the mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation and clinical signs of mitochondrial disease - The KHENERGY study | Inherited mitochondrial disease, including MELAS (mitochondrial Encephalopathy Lactic Acidosis and Stroke like episodes) and MIDD (Maternally Inherited Diabetes and Deafness);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: KH176 Product Code: KH176 | Khondrion BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Netherlands | |||
| 2 | NCT02909400 (ClinicalTrials.gov) | September 2016 | 13/9/2016 | The KHENERGY Study | An Exploratory, Double-blind, Randomized, Placebo-controlled, Single-center, Two-way Cross-over Study With KH176 in Patients With the Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation and Clinical Signs of Mitochondrial Disease | Mitochondrial Diseases;Mitochondrial Myopathies;Mitochondrial Encephalomyopathies;MELAS;MIDD | Drug: KH176;Drug: placebo | Khondrion BV | Radboud University | Completed | 18 Years | N/A | All | 20 | Phase 2 | Netherlands |
| 3 | NCT02544217 (ClinicalTrials.gov) | May 2015 | 19/4/2015 | A Dose-escalating Clinical Trial With KH176 | A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176 | MELAS;LHON;Leigh Syndrome;Mitochondrial Disease;Mitochondrial DNA tRNALeu(UUR) m.3243A| Drug: KH176;Drug: placebo | Khondrion BV | Drug Research Unit Ghent, Belgium | Completed | 18 Years | 55 Years | Male | 32 | Phase 1 | Belgium |
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| 4 | 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 |