Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients
- Conditions
- Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 yearsMedDRA version: 9.1Level: LLTClassification code 10032950
- Registration Number
- EUCTR2010-020386-24-IT
- Lead Sponsor
- TROPHOS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 150
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.MFM relative score (percentage of the maximum sum of both dimensions)= 15% (D1 + D2) 4.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 then 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 parent/guardian 9.Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities 10.Ability to take study treatment (tested at screening after informed consent)
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1.Evidence of renal dysfunction,blood dysplasia,hepatic insufficiency,symptomatic pancreatitis,congenital heart defect,known hystory of metabolic acidosis,hypertension,significant central nervous system impairment,or neurodegenerative or neuromuscular disease other then 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 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,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 ß-agonists (for the treatment of asthma crisis per example)is allowed 5.Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of it 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 medication 8.Olesoxime is contraindicated in subjects who develop drug hypersensitivity to it or one of the formulaton excipients including hypersensitivity to sesame oil 9.Patients with hemostasis disorders 10.patients with known biliary tract obstruction 11.Current or planned pregnancy or nursing period 12.For woman:failure to use one of the following safe methods of contraception-female condoms,diaphragm or coil,each used in combination with spermicides;intra-uterine device;hormonal contraception in combination with a mechanical method of contraception 13.participation in any other investigational drug or therapy study within the previous 3 months.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 in non ambulant patients aged 3-25 years;Secondary Objective: ;Primary end point(s): The primary outcome measure is the Motor Function Measure (MFM)D1+D2 score
- Secondary Outcome Measures
Name Time Method