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

Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS

Status

Active, not recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001589-25-GB

Enrollment

173

Registered

2015-08-04

Start date

2015-11-19

Completion date

Unknown

Last updated

2020-07-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal Muscular Atrophy MedDRA version: 20.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital System Organ Class: 100000004850

Interventions

Product Name: Olesoxime Product Code: RO7090919 Pharmaceutical Form: Powder and solvent for oral suspension INN or Proposed INN: OLESOXIME CAS Number: 22033-87-0 Current Sponsor code: RO7090919/F01 Co

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
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 • To evaluate the disease associated medical complications and procedures in olesoxime treated patients compared to the natural history of disease • To evaluate the disease course between last visit of the studies TRO19622CLEQ1275-1 and TRO19622CLEQ1115-1, and baseline assessment in this study • To investigate the pharmacokinetics (PK) of olesoxime in the target population • To explore changes in level of independence and health-related quality of life following treatment with olesoxime • To assess health-related quality of life and conduct economic modeling using the EuroQol 5-Dimension, 5-Level Questionnaire (EQ-5D-5L);Primary end point(s): 1. Incidence of adverse events 2. Safety laboratory tests, Vital signs and ECG;Timepoint(s) of evaluation of this end point: 1. Up to 4 years 2. Up to 4 years	—

Measure

Time frame

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 • To evaluate the disease associated medical complications and procedures in olesoxime treated patients compared to the natural history of disease • To evaluate the disease course between last visit of the studies TRO19622CLEQ1275-1 and TRO19622CLEQ1115-1, and baseline assessment in this study • To investigate the pharmacokinetics (PK) of olesoxime in the target population • To explore changes in level of independence and health-related quality of life following treatment with olesoxime • To assess health-related quality of life and conduct economic modeling using the EuroQol 5-Dimension, 5-Level Questionnaire (EQ-5D-5L);Primary end point(s): 1. Incidence of adverse events 2. Safety laboratory tests, Vital signs and ECG;Timepoint(s) of evaluation of this end point: 1. Up to 4 years 2. Up to 4 years

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Secondary

Measure	Time frame
Secondary end point(s): 1. Change from baseline in Motor Function Measure (MFM) D1+D2 score and total score 2. Predose (trough) plasma olesoxime concentration at each visit 3. The Pediatric Quality of Life Inventory (PedsQL) core scales and neuromuscular sub-scales 4. The EuroQol 5-Dimension Questionnaire, 5-level version (EQ-5D-5L) score 5. SMA Independence Scale (SMAIS);Timepoint(s) of evaluation of this end point: 1. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 2. Baseline (Week 1), Week 13, Week 26, Week 39, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 3. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 4. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 5. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study	—

Measure

Time frame

Secondary end point(s): 1. Change from baseline in Motor Function Measure (MFM) D1+D2 score and total score 2. Predose (trough) plasma olesoxime concentration at each visit 3. The Pediatric Quality of Life Inventory (PedsQL) core scales and neuromuscular sub-scales 4. The EuroQol 5-Dimension Questionnaire, 5-level version (EQ-5D-5L) score 5. SMA Independence Scale (SMAIS);Timepoint(s) of evaluation of this end point: 1. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 2. Baseline (Week 1), Week 13, Week 26, Week 39, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 3. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 4. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study 5. Baseline (Week 1), Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 6 months until end of study

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Countries

Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom

Contacts

Public ContactTrial Information Support Line-TISL

F. Hoffmann-La Roche Ltd

global.rochegenentechtrials@roche.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026