A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

• Status: Completed
• Trial end date: 2018-12-18
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (open-label Phase Ib, multicenter, single- and multiple- dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II/III, adaptive, parallel-group, double blind, randomized, placebo-controlled, multicenter, multinational study).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche

Criteria

Inclusion Criteria:

• Participation in the previous studies (TRO19622 CL E Q 1115-1 or TRO19622 CL E Q 1275-1)

• 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

Exclusion Criteria:

• Female participants who are pregnant or lactating, or intending to become pregnant during the study

• Participants who, in the opinion of the investigator, are not suitable to participate in this open-label study

• Participants who have developed study drug hypersensitivity to olesoxime or one of the formulation excipients, including sesame oil

• Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening

• Concomitant or previous participation in a survival motor neuron 2 (SMN2) targeting antisense oligonucleotide study within 6 months prior to screening

• History of human immunodeficiency virus infection, history of Hepatitis B infection within the past year, history of Hepatitis C infection which has not been adequately treated

• History of illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment

• Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study

• History or presence of an abnormal electrocardiogram (ECG) that is clinically significant in the investigator's opinion