Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
NCT ID: NCT01302600
Last Updated: 2016-11-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
165 participants
INTERVENTIONAL
2010-11-30
2013-10-31
Brief Summary
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Detailed Description
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Stage 1 DMC 3-month safety assessment: An independent Data Monitoring Committee (DMC)will assess the safety of olesoxime every 3 months.
Stage 2 Efficacy/futility analyses at one year: A first interim efficacy analysis will be performed after all patients have been treated for one year (52 weeks) in order to assess the need to continue the study to reach the planned objective. In the event of positive and significant results in favor of olesoxime, the study will be considered as successful and all patients will be switched to olesoxime to allow the assessment of the sustainability of the treatment effect and safety. If the results are significantly in favor of placebo, the study will be discontinued for failure (futility).
Stage 3 Efficacy and safety analysis at two years: The expected study duration is of 2 years (104 weeks) to show efficacy. If the study is not discontinued for futility or medication regimen is changed due to success, the study will therefore continue until planned completion i.e. 104 weeks.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Olesoxime
100 patients in this arm. liquid suspension
Olesoxime
Liquid suspension formulation, 100 mg/ml at a dose of 10 mg/kg will be administered once a day with food at dinner
Placebo
50 patients enrolled in this arm. liquid suspension
Placebo
0.1ml/kg once a day with food at dinner.
Interventions
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Olesoxime
Liquid suspension formulation, 100 mg/ml at a dose of 10 mg/kg will be administered once a day with food at dinner
Placebo
0.1ml/kg once a day with food at dinner.
Eligibility Criteria
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Inclusion Criteria
* Laboratory documentation of homozygous absence of SMNI exon 7 and/or deletion and mutation on other allele
* MFM relative score (percentage of the maximum sum of both dimensions) \>= 15% (D1 + D2 score)
* HFMS score at baseline \>= 3
* Non ambulant patients defined as patients with HFMS score =\< 38
* Must be 3 years of age or older, but younger than 26 years of age, at time of enrolment
* Age of onset of symptoms =\< 3 years of age
* Signed informed consent of patient and/or parents/guardian
* Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities
* Ability to take the study treatment (tested at screening after informed consent)
Exclusion Criteria
* Any clinically significant ECG abnormality
* Any acute co-morbid condition interfering with the well-being of the subject within 7 days of enrolment including bacterial infection, viral infectious processes, food poisoning, temperature \> 37.0 °C, the need for acute treatment or observation due to any other reason, as judged by the investigator; patient can be included after resolution of the acute event
* Use of medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, agents anticipated to increase or decrease muscle strength or agents with known or presumed histone deacetylase (HDAC) inhibition, within 30 days prior to study entry. Subjects who use a nebulizer or require an inhaler to steroids will be allowed in the study; however oral use of steroids is prohibited. The oral use of salbutamol is permitted with the following restrictions: patients should have been on salbutamol for at least 6 months before inclusion in the trial, with good tolerance. The dose of salbutamol should remain constant for the duration of the trial. The use of inhaled beta-agonists (for the treatment of asthma crisis for example) is allowed.
* Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of rod or fixation within 6 months of enrolment.
* Inability to meet study visit requirements or cooperate reliably with functional testing
* Coexisting medical conditions that contraindicate travel, testing or study medications
* Olesoxime is contraindicated in subjects/patients who develop drug hypersensitivity to it or one of the formulation excipients including hypersensitivity to sesame oil.
* Patients with hemostasis disorders
* Patients with known biliary tract obstruction
* Current or planned pregnancy or nursing period
* For Women: Failure to use one of the following safe methods of contraception:
1. Female condoms, diaphragm or coil, each used in combination with spermicides
2. Intra-uterine device
3. Hormonal contraception in combination with a mechanical method of contraception
* Participation in any other investigational drug or therapy study within the previous 3 months.
3 Years
25 Years
ALL
No
Sponsors
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Association Française contre les Myopathies (AFM), Paris
OTHER
Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Enrico Bertini, MD
Role: PRINCIPAL_INVESTIGATOR
Bambino Gesu Hospital
Locations
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University Hospitals
Ghent, , Belgium
University Hospitals
Leuven, , Belgium
Hôpital Femme-Mère-Enfant
Bron, , France
Hôpital Raymond Poincaré
Garches, , France
CHRU Hôpital R. Salengro
Lille, , France
Hôpital La Timone
Marseille, , France
CHU de Montpellier, Hôpital Gui de Chauliac
Montpellier, , France
Groupe hospitalier Armand-Trousseau
Paris, , France
University of Essen
Essen, , Germany
Universitat Klinikum Freiburg
Freiburg im Breisgau, , Germany
Friedrich-Baur-Institute
München, , Germany
Istituto Giannina Gaslini
Genova, , Italy
AOU Policlinico
Messina, , Italy
Centro Dino Ferrari, Milano
Milan, , Italy
NEuroMuscular Omnicentre (NEMO)
Milan, , Italy
Bambino Gesu' Research Children's Hospital
Roma, , Italy
Policlinico Universitario "Agostino Gemelli",
Roma, , Italy
University Medical Center
Utrecht, , Netherlands
Medical University of Warsaw
Warsaw, , Poland
Birmingham Heartlands Hospital
Birmingham, , United Kingdom
Dubowitz Neuromuscular Centre
London, , United Kingdom
Institute of Human Genetics
Newcastle, , United Kingdom
Countries
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References
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Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators. Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Neurol. 2017 Jul;16(7):513-522. doi: 10.1016/S1474-4422(17)30085-6. Epub 2017 Apr 28.
Other Identifiers
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TRO19622 CL E Q 1275-1
Identifier Type: OTHER
Identifier Source: secondary_id
WN29836
Identifier Type: -
Identifier Source: org_study_id