Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA
- Conditions
- Disease of muscles and nerves100283961002830210029317
- Registration Number
- NL-OMON43897
- Lead Sponsor
- Roche Nederland B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 9
* 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 last dose of olesoxime.
* 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Due to progressive proximal muscle weakness, study TRO19622CLEQ1275-1<br /><br>considered the MFM D1 + D2 score as the most appropriate measure in patients<br /><br>with SMA Type 2 and non-ambulatory Type 3 and as such it was used as the<br /><br>primary endpoint in the study. As this open-label study aims to provide further<br /><br>clinical information and complement the results of study TRO19622CLEQ1275-1<br /><br>consistency in primary endpoint is appropriate. The primary endpoint is<br /><br>therefore MFM D1 + D2.</p><br>
- Secondary Outcome Measures
Name Time Method