MedDRA - 12.1 LLT 10041582 Spinal Muscular Atrophy G12
Conditions
Interventions
Group 1: Placebo: matching liquid suspension formulation administred once a day
Group 2: Olesoxime: suspension 10mg/ml administered at 10mg/kg/day ( once a day)
Sponsors
TROPHOS SA
Eligibility
Sex/Gender
All
Age
3 Years to 25 Years
Inclusion criteria
Inclusion criteria: •Weakness and hypotonia consistent with a clinical diagnosis of spinal muscular atrophy (SMA) type II or III •Laboratory documentation of homozygous absence of SMNI exon 7 and/or deletion and mutation on other allele •MFM relative score (percentage of the maximum sum of both dimensions) >= 15% (D1 + D2 score) •HFMS score at baseline >= 3 •Non ambulant patients defined as patients with HFMS score =< 38 •Must be 3 years of age or older, but younger than 26 years of age, at time of enrolment •Age of onset of symptoms =< 3 years of age •Signed informed consent of patient and/or parents/guardian •Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities •Ability to take the study treatment (tested at screening after informed consent)
Exclusion criteria
Exclusion criteria: •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 •Any clinically significant ECG abnormality •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 •Use of medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, 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 beta-agonists (for the treatment of asthma crisis for example) is allowed. •Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of rod or fixation within 6 months of enrolment. •Inability to meet study visit requirements or cooperate reliably with functional testing •Coexisting medical conditions that contraindicate travel, testing or study medications •Olesoxime is contraindicated in subjects/patients who develop drug hypersensitivity to it or one of the formulation excipients including hypersensitivity to sesame oil. •Patients with hemostasis disorders •Patients with known biliary tract obstruction •Current or planned pregnancy or nursing period •For Women: Failure to use one of the following safe methods of contraception: 1.Female condoms, diaphragm or coil, each used in combination with spermicides 2.Intra-uterine device 3.Hormonal contraception in combination with a mechanical method of contraception •Participation in any other investigational drug or therapy study within the previous 3 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Motor Fonction Measure (MFM) D1+D2 score The Motor Function Measure will be evaluated at Day 0, 6 months (week 26), 1 year (week 52),18 months (week 78) and 2 years (week 104)/end of study. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary outcome measures will be responder analyses on MFM and HFMS, time to 4 point decrease on HFMS, CMAP/MUNE, PedsQL, FVC, CGI and Safety . - HMFS will be measured at screening period, 3 months (week 13), 9 months (week 39), 15 months (week 65) and 21 months (week 91) - CMAP/MUNE :electromyography measures CMAP followed by a MUNE assessment using the modified incremental method, will be measured at Day 0, 6 months (week 26), 1 year (week 52), 18 months (week 78) and 2 years (week 104)/end of study. - PedsQL : Pediatric quality of life .PedsQL will be evaluated at Day 0, 1 year (week 52) and at 2 years (week 104)/end of study. - FVC (forced vital capacity): pulmonary function .The forced vital capacity will be evaluated every 3 months during the first year and every 6 months thereafter - CGI :Clinical global impression (scale)The Clinical Global Impression scale will be assessed at Day 0, 3 months (week 13), 6 months (week 26), 9 months (week 39), 1 year (week 52), 15 months (week 65), 18 months (week 78), 21 months (week 91) and 2 years (week 104) both and separately by patients/ parents and physicians and at 2 months (week 9) only by patients/ parents. | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom
Contacts
Public ContactIsabel Clémançon de Bellefois
TROPHOS S.A
Outcome results
None listed