Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA
- Conditions
- Spinal Muscular AtrophyMedDRA version: 18.0Level: LLTClassification code 10051203Term: Spinal muscular atrophy congenitalSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2015-001589-25-IT
- Lead Sponsor
- F. Hoffmann-La Roche Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot 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
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method