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The MitoTarget Trial
MITOTARGET: Clinical Study of Safety and Efficacy of TRO19622 as Add-on Therapy to Riluzole versus Placebo in Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)
Disease
Amyotrophic Lateral Sclerosis (ALS) /
or Motor Neuron Disease (UK) /
or Maladie de Charcot (France)
Study Summary
The main aim of the next trial is to see whether TRO19622 (olesoxime) can improve the survival of patients with ALS.
TRO19622 is a molecule discovered by Trophos, the company heading the MitoTarget Consortium, that has a cholesterol-like structure and displays strong neuroprotective properties. TRO19622 is as effective as a cocktail of three neurotrophic factors in keeping motor neurons alive in culture.
The study will also generate important information regarding the safety, tolerability and plasma levels (the concentration in the blood) of TRO19622-olesoxime in patients with ALS.
This study will investigate whether a single daily dose of two capsules of this new drug, given every day at lunchtime, and administered over a treatment period of 18 months, can improve survival and symptoms of ALS patients.
The trial comprises a screening test and a treatment phase. Patients will be seen by their doctors every month for the first three months of treatment and then every three months thereafter. At the end of the treatment period, patients will have the opportunity to be enrolled in an open-label safety study that will be conducted under a separate protocol.
Enrolled patients will be participating in a research that is part of a European project run by the MitoTarget Consortium involving sites in France, Belgium, Germany, the UK and Spain. The study started in May 2009 and results of the study are expected in mid 2011.
Information on the partners within the MitoTarget Consortium can be found here
The new drug that the members of the consortium are studying in this clinical trial, TRO19622-olesoxime, is a substance that has so far not been granted final approval for treatment of ALS or any other diseases. It is a substance that is derived from a natural metabolite of cholesterol and which was found to exert neuroprotective and neuroregenerative (restoring) effects on nerve cells in various cell culture and animal models.
TRO19622-olesoxime is currently being developed for the treatment of two motor neuron diseases: Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). It is also being explored for the treatment of painful Chemotherapy Induced Peripheral Neuropathy (CIPN).
So far, TRO19622-olesoxime has been investigated in 5 clinical studies in healthy volunteers and in a one month of safety and tolerance trial in patients with ALS. The results of the previous studies demonstrated that TRO19622-olesoxime is generally well-tolerated.
