A study testing olesoxime as a treatment for spinal muscular atrophy type 2 and 3

Start Date
01/11/2010
Expected end Date
01/11/2013
Location
Europe
Status
Ongoing but has finished recruiting patients

What is the aim of the trial?

This Phase 2 clinical trial aims to establish if a drug called olesoxime is a safe and effective treatment for spinal muscular atrophy type 2 and 3. Phase 2 trials are designed to test how well the drug or treatment works as well as to continue safety assessments on a larger group of patients.

Spinal muscular atrophy is a genetic condition that results in progressive wasting and weakness of the muscles in the arms and legs of infants, children and adults. This muscle wasting is the result of a shortage in a protein called ‘survival motor neuron’ (SMN), due to mistakes or “mutations” in the gene which contains the instructions for making it.

SMN protein is essential for the survival of motor neurons, which are the spinal cord cells that connect with the nerves and control muscle movement. Without enough SMN protein, motor neurons are gradually lost.

Studies carried out on cells grown in a lab and in mice have shown that olesoxime (previously referred to as TRO19622), protects neurons and helps them to survive. Olesoxime has successfully completed phase 1 clinical trials (in healthy volunteers and adults with another condition), which showed the drug was safe and well tolerated. These studies also determined what dose to use in further trials. Based on results obtained in mice, it is hoped that olesoxime could potentially be used to reduce progression of weakness in individuals with spinal muscular atrophy.

Who can be involved in the trial?

The trial expects to recruit 150 participants with spinal muscular atrophy type 2 or 3, aged between three and 25, in seven European countries. Participants must be unable to walk unassisted and have had symptoms of spinal muscular atrophy since before they were three years of age.

Some kidney, liver or heart problems, the use of certain medications and participation in other clinical trials may make patients ineligible from the study. Pregnancy or breastfeeding will also make women ineligible for the trial.

What happens during the trial?

Participants will be randomly assigned to take olesoxime (at a dose of 10mg per kg of body weight) or a placebo (at a dose of 0.1mg per kg), once a day as a liquid suspension with food at dinner for two years. Neither the patients nor the clinicians will know who is receiving the placebo and who is receiving olesoxime.

The safety of olesoxime will be checked every three months with a series of blood tests and other tests such as ECG (electrical testing of the heart function) and the effectiveness measured every six months, by assessing muscle function.

The study is planned to last for two years but after the first year the preliminary results will be analysed. If the results are very positive, then all patients who were taking the placebo will receive olesoxime instead. If however after one year the results show olesoxime is no more effective than the placebo, the trial will be stopped. If the results are inconclusive after the first year the study will continue for the full two years.

Where is the study taking place?

The study is taking place in:
•        United Kingdom: London, Birmingham and Newcastle upon Tyne (London and Birmingham will only recruit children, while Newcastle will also recruit adults)
•        Belgium: Ghent and Leuven
•        France: Bron, Garches, Lille, Marseille, Montpellier and Paris
•        Germany: Essen, Freiburg and Munchen
•        Italy: Genova, Messina, Milan and Rome
•        Netherlands: Utrecht
•        Poland: Warsaw

In most circumstances, for somebody to participate in a clinical trial, they need to live near the team of people who are conducting the research, because they need to be closely monitored.

How could the results of the trial benefit patients?

The results from this trial will determine if olesoxime, when taken once-a-day, is a safe and effective treatment for spinal muscular atrophy. Further trials may then be needed to test the long term safety and effectiveness in more patients. If this clinical trial is successful, olesoxime could potentially be used to delay, or even stop further progression of spinal muscular atrophy.

Contact Details:

Ms Hinal Patel

Study coordinator associated to the Principal Investigator (F. Muntoni)

Tel: +44 (0)20 7905 2639    

Email: hinal.patel@ucl.ac.uk

Who is funding this study?

  • French Association against Myopathies (AFM)

Official name of the Trial:

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients

Trial study number:

NCT01302600

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

http://clinicaltrials.gov/ct2/show/NCT01302600

Background information and related links:

This Phase 2 clinical trial aims to establish if a drug called olesoxime is a safe and effective treatment for spinal muscular atrophy type 2 and 3. Phase 2 trials are designed to test how well the drug or treatment works as well as to continue safety assessments on a larger group of patients.

Spinal muscular atrophy is a genetic condition that results in progressive wasting and weakness of the muscles in the arms and legs of infants, children and adults. This muscle wasting is the result of a shortage in a protein called ‘survival motor neuron’ (SMN), due to mistakes or “mutations” in the gene which contains the instructions for making it.

SMN protein is essential for the survival of motor neurons, which are the spinal cord cells that connect with the nerves and control muscle movement. Without enough SMN protein, motor neurons are gradually lost.

Studies carried out on cells grown in a lab and in mice have shown that olesoxime (previously referred to as TRO19622), protects neurons and helps them to survive. Olesoxime has successfully completed phase 1 clinical trials (in healthy volunteers and adults with another condition), which showed the drug was safe and well tolerated. These studies also determined what dose to use in further trials. Based on results obtained in mice, it is hoped that olesoxime could potentially be used to reduce progression of weakness in individuals with spinal muscular atrophy.

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