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    Summary
    EudraCT Number:2015-001589-25
    Sponsor's Protocol Code Number:BN29854
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-10-07
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2015-001589-25
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy
    A.3.2Name or abbreviated title of the trial where available
    OLEOS
    A.4.1Sponsor's protocol code numberBN29854
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorF. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/05/264
    D.3 Description of the IMP
    D.3.1Product nameOlesoxime
    D.3.2Product code RO7090919
    D.3.4Pharmaceutical form Powder and solvent for oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOLESOXIME
    D.3.9.1CAS number 22033-87-0
    D.3.9.2Current sponsor codeRO7090919/F01
    D.3.9.4EV Substance CodeSUB32715
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number7.5
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSESAME OIL, REFINED
    D.3.9.2Current sponsor codeRO7090919/F02
    D.3.9.3Other descriptive nameSESAME OIL, REFINED
    D.3.9.4EV Substance CodeSUB12275MIG
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number67.1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Yes
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Spinal Muscular Atrophy
    E.1.1.1Medical condition in easily understood language
    A neuromuscular, genetic disorder clinically characterized by progressive muscular weakness and atrophy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10051203
    E.1.2Term Spinal muscular atrophy congenital
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To evaluate the safety of olesoxime in patients with Spinal Muscular Atrophy (SMA)
    E.2.2Secondary objectives of the trial
    • 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)
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence of adverse events
    2. Safety laboratory tests, Vital signs and ECG
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 4 years
    2. Up to 4 years
    E.5.2Secondary 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)
    E.5.2.1Timepoint(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.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic Yes
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The study will continue until olesoxime is commercially available in the patient’s country, or as per local regulation, or per the Sponsor’s decision to terminate the olesoxime program for SMA, but will not exceed 4 years after the last patient was enrolled in the study.
    NB In the UK, the study will last for a fixed period of 3 years.
    The end of this study is defined as the date when the last patient, last visit (LPLV) of the treatment period occurs
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 117
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 69
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 48
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 56
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    The study doesn't exclude such populations, inclusion/continuation will be decided upon discussion between patient and physician.
    Patients incapable of consent (i.e. minors) will provide assent and their caregiver, consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 173
    F.4.2.2In the whole clinical trial 173
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    The Sponsor will offer post-trial access to the study drug olesoxime free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product

    The Roche Global Policy on Continued Access to Investigational Medicinal Product is available at the following Web site:
    http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-12-18
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