A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy

Phase 1

• Conditions: Spinal Muscular Atrophy; MedDRA version: 20.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-001589-25-NL
• Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-10-07
• Study Completion: 2018-12-18
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 131

Inclusion Criteria

• 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

Exclusion Criteria

• 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