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

• Register: EUCTR
• Last refreshed on: 20 July 2020
• Main ID: EUCTR2015-001589-25-GB
• Date of registration: 04/08/2015
• Prospective Registration: Yes
• Primary sponsor: F. Hoffmann-La Roche Ltd
• Public title: A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy
• Scientific title: 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
• Date of first enrolment: 19/11/2015
• Target sample size: 173
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-001589-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Trial Information Support Line-TISL
• Affiliation: F. Hoffmann-La Roche Ltd

• Name: Trial Information Support Line-TISL
• Affiliation: F. Hoffmann-La Roche Ltd

Key inclusion & exclusion 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:
• 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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]

Intervention(s)

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
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 7.5-
INN or Proposed INN: SESAME OIL, REFINED
Current Sponsor code: RO7090919/F02
Other descriptive name: SESAME OIL, REFINED
Concentration unit: ml millilitre(s)
Concentration type: equal
Concentration number: 67.1-

Primary Outcome(s)

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

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)

Main Objective: • To evaluate the safety of olesoxime in patients with Spinal Muscular Atrophy (SMA)

Secondary Outcome(s)

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

Secondary ID(s)

BN29854

Source(s) of Monetary Support

F. Hoffmann-La Roche Ltd

Ethics review

Status: Approved
Approval date: 19/11/2015
Contact:

Results

• Results available: Yes
• Date Posted: 22/06/2019
• Date Completed: 18/12/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001589-25/results