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August 2019

The number of clinical trials taking place around the world is slowly increasing, giving renewed hope to patients. The trials are focused on a range of mitochondrial disorders.

KH176, a promising oral small molecule is in Phase IIb of clinical development in the Netherlands. Owned by Khondrion, the compound aims to treat MELAS, Leigh disease, inherited mitochondrial respiratory chain disorders and maternally inherited diabetes and deafness caused by a mutation within mitochondrial DNA. Read more about this trial.

Another potential treatment of inherited mitochondrial respiratory chain diseases is undergoing it’s second early clinical trial (Phase 1a/b) in the UK. The molecule, KL1333, is being developed by NeuroVive to treat primary genetic mitochondrial disorders such as: mitochondrial encephalomyopathy, lactic acidosis and stroke-like episode (MELAS), Kearns-Sayre syndrome (KSS), chronic progressive external ophthalmoplegia (CPEO), Pearson syndrome, and myoclonic epilepsy with ragged red fibres (MERRF) syndrome. Read more about this trial.

A Phase II trial in Barth syndrome patients investigating the use of cholesterol medication opened in the UK in July. The CARDIOMAN clinical trial will evaluate whether cholesterol drug, bezafibrate can treat men and boys with Barth syndrome, a type of mitochondrial disease (mito).  Read more about this trial.

A second trial in Barth syndrome is underway in the USA, investigating injections of elamipretide. This Phase III trial by Stealth BioTherapeutics aims to develop elamipretide as a potential treatment for mito. Read more about this trial.

A Phase III clinical trial of GS010, GenSight Biologics’ gene therapy, is showing encouraging results with Leber hereditary optic neuropathy (LHON) patients. The trial is designed to assess the efficacy and safety of GS010 in improving the visual acuity in patients from the USA, Europe and Taiwan. Read more about this trial.

The first mitochondrial cell therapy trial to treat a mitochondrial disease is taking place in Israel. The Mitochondrial Augmentation Therapy aims to increase the levels of normal mitochondrial DNA in paediatric Pearson syndrome patients. The Minovia Therapeutics trial is in Phase I/II. Read more about this trial.

This article highlights just a few of the growing number of trials taking place around the world. It can take years for new drugs to reach patients, but each new trial brings us closer to the production of effective treatments and cures that are urgently needed to improve and ultimately save lives.

Also see our page on Studies Taking Place in the World.


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