A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy
- Conditions
- Spinal Muscular AtrophyMedDRA version: 18.1Level: LLTClassification code 10051203Term: Spinal muscular atrophy congenitalSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2015-001589-25-FR
- Lead Sponsor
- F. Hoffmann-La Roche Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- All
- Target Recruitment
- 131
• Participation in the previous studies (TRO19622CLEQ11150-1 or TRO19622CLEQ1275-1)
• Able to comply with the study protocol, in the investigator’s judgment, including ability to take study treatment and perform study visits
• For women of childbearing potential: agreement to use an acceptable birth control method during the treatment period and for at least 28 days after the last dose of olesoxime.
Are the trial subjects under 18? yes
Number of subjects for this age range: 117
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 56
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
• Patients who, in the opinion of the investigator, are not suitable to participate in this open label study
• Patients who have developed study drug hypersensitivity to olesoxime or one of the formulation excipients, including sesame oil
• Concomitant or previous participation in any other investigational drug or device study within 90 days prior to screening
• Concomitant or previous participation in a survival motor neuron protein gene (SMN2) targeting antisense oligonucleotide study within 6 months prior to screening
• Pregnant or lactating, or intending to become pregnant during the study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: • To evaluate the safety of olesoxime in patients with Spinal Muscular Atrophy (SMA) ;Secondary Objective: • To evaluate effectiveness of olesoxime compared to the natural history of disease in patients with SMA<br>• To evaluate the disease associated medical complications and procedures in olesoxime treated patients compared to the natural history of disease<br>• To evaluate the disease course between last visit of the studies TRO19622CLEQ1275-1 and TRO19622CLEQ1115-1, and baseline assessment in this study<br>• To investigate the pharmacokinetics (PK) of olesoxime in the target population <br>• To compare changes in health related quality of life (QOL) and the relationship to other measures of disease status, post olesoxime treatment versus the natural history of disease in patients with SMA;Primary end point(s): 1. Incidence of adverse events<br>2. Safety laboratory tests, Vital signs and ECG;Timepoint(s) of evaluation of this end point: 1. Up to 4 years<br>2. Up to 4 years
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Change from baseline in Motor Function Measure (MFM) D1+D2 score and total score<br>2. Change from baseline in Hammersmith Functional Motor Scale (HFMS) score<br>3. Predose (trough) plasma olesoxime concentration at each visit<br>4. The Pediatric Quality of Life Inventory (PedsQL) core scales and neuromuscular sub-scales<br>5. The EuroQol 5-Dimension Questionnaire, 5-level version (EQ-5D-5L) score;Timepoint(s) of evaluation of this end point: 1. Baseline (Week 1), Week 26, Week 52, Week 104, thereafter every 12 months until end of study<br>2. Screening (Day -42 to Day -1), Week 13, Week 39, Week 78, Week 130, thereafter every 12 months until end of study<br>3. Week 1, Week 13, Week 26, Week 52, Week 104, thereafter every 12 months until end of study<br>4. Week 1, Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 26 weeks until end of study<br>5. Week 1, Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 26 weeks until end of study