With the support of the afm and the involvement of clinicians in finding new treatments, the company trophos continues clinical development of a molecule called olesoxime (tro 19622) in spinal muscular atrophy (sma) type ii and iii

Thanks to the support from the French Muscular Dystrophy Association (AFM) and the involvement of clinicians in the search for new treatments, Trophos is developing olesoxime (TRO19622) for treatment of spinal muscular atrophy (SMA). Previous Phase I clinical trials in children and young adults showed that olesoxime is well absorbed and well tolerated. A phase II clinical trial will start recruiting soon in several European countries, including the United Kingdom with the objective to assess efficacy and the safety of olesoxime in 150 patients with type II and III SMA. This clinical trial concerns non-ambulant patients from 3 to 25 years old. The treatment procedure will last for 24 months and involves periodic visits to the medical centre. Please find the following list of inclusion and exclusion criteria you should take into account for this trial: Inclusion criteria: 1. Weakness and hypotonia consistent with a clinical diagnosis of spinal muscular atrophy (SMA) type II or III 2. Laboratory documentation of homozygous absence of SMNI exon 7 and/or deletion and mutation on other allele 3. Motor Function Measure relative score (percentage of the maximum sum of both dimensions) ≥ 15% (D1+ D2 score) 4. Hammersmith Functional Motor Scale (HFMS) score at baseline ≥ 3 5. Non ambulant patients defined as patients with HFMS score ≤ 38 6. Must be 3 years of age or older, but younger than 26 years of age, at time of enrolment 7. Age of onset of symptoms ≤ 3 years of age 8. Signed informed consent of patient and/or parents/guardian 9. Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities 10. Ability to take the study treatment (tested at screening after informed consent) Exclusion criteria: Subjects who meet one or more of the following criteria are not allowed to be included in the study: 1. Evidence of renal dysfunction, blood dysplasia, hepatic insufficiency, symptomatic pancreatitis, congenital heart defect, known history of metabolic acidosis, hypertension, significant central nervous system impairment, or neurodegenerative or neuromuscular disease other than SMA 2. Any clinically significant ECG abnormality 3. Any acute co-morbid condition interfering with the well-being of the subject within 7 days of enrolment including bacterial infection, viral infectious processes, food poisoning, temperature > 37.0 °C, the need for acute treatment or observation due to any other reason, as judged by the investigator; patient can be included after resolution of the acute event 4. Use of medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, agents anticipated to increase or decrease muscle strength or agents with known or presumed histone deacetylase (HDAC) inhibition, within 30 days prior to study entry. Subjects who use a nebulizer or require an inhaler to steroids will be allowed in the study; however oral use of steroids is prohibited. The oral use of salbutamol is permitted with the following restrictions: patients should have been on salbutamol for at least 6 months before inclusion in the trial, with good tolerance. The dose of salbutamol should remain constant for the duration of the trial. The use of inhaled agonists (for the treatment of asthma crisis for example) is allowed. 5. Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of rod or fixation within 6 months of enrolment 6. Inability to meet study visit requirements or cooperate reliably with functional testing 7. Coexisting medical conditions that contraindicate travel, testing or study medications 8. Olesoxime is contraindicated in subjects/patients who develop drug hypersensitivity to it or one of the formulation excipients including hypersensitivity to sesame oil. 9. Patients with hemostasis disorders 10. Patients with known biliary tract obstruction If you meet the above criteria, you may be able to participate to this trial. For this, please contact one of the 3 centres of the United Kingdom. Investigators will answer your questions (course, eligibility criteria, expected benefits, potential risks .) and verify, in conjunction with your physician, that you meet the eligibility criteria. Contact details of the centres are: -Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, Dr K. Ganeshaguru, phone number: +44 20 7905 2151 -Institute of Genetic Medicine, Newcastle upon Tyne, Prof. Hanns Lochmüller, phone number: +44 191 241 8757 -Birmingham Heartlands Hospital, Birmingham, Dr Helen Roper, phone number: +44 121 424 3687 Please note that the London and Newcastle clinical trial sites are now open, while the Birmingham site should open in the coming weeks. With best wishes. Yours sincerely, Francesco Muntoni (Country Principal Investigator)

Source: http://www.jtsma.org.uk/res/invitation%20letter%20.pdf