Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS (MITOTARGET)
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|ClinicalTrials.gov Identifier: NCT00868166|
Recruitment Status : Completed
First Posted : March 24, 2009
Results First Posted : February 15, 2019
Last Update Posted : February 28, 2019
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Olesoxime Drug: Placebo Comparator Drug: Riluzole||Phase 3|
A stand alone treatment with TRO19622 is not acceptable for ethical reasons. Riluzole is an approved and widely used ALS treatment in the European community, in Japan and in the USA.
Therefore, in this study, TRO19622 will be assessed as add-on to riluzole in patients suffering from ALS.
At the start of the study, patients will be randomized to one of two groups : TRO19622 (330 mg QD or placebo (once a day).
Each treatment will be administered for 18 months under double-blind conditions. The product under evaluation will be administered to patients receiving the standard of care for ALS, including riluzole.
Riluzole dosage (50 mg bid) must be stable and well tolerated for at least one month prior to inclusion into the study.
After the double-blind period, open-label administration of TRO19622 will be allowed for safety and survival assessments and until efficacy results are available.
A separate open-label protocol will be written 6 months after the randomization of the last patient into the study.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||512 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Phase II/III, Multicenter, Randomized, Parallel Group, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) Patients Treated With Riluzole|
|Actual Study Start Date :||April 30, 2009|
|Actual Primary Completion Date :||September 30, 2011|
|Actual Study Completion Date :||September 30, 2011|
2 Capsules of TRO19622 (330mg) once a day with the noon meal as add-on therapy to riluzole 50mg bid
2 capsules of TRO19622 (330mg) once a day with the noon meal as add-on therapy to riluzol 50mg bid
Other Name: TRO19622
Riluzole given as add-on therapy 50mg bid
Other Name: Rilutek
Placebo Comparator: Placebo Comparator
2 Capsules of Placebo once a day with the noon meal as add-on therapy to riluzole 50mg bid
Drug: Placebo Comparator
2 capsules of Placebo once a day with the noon meal as add-on therapy to riluzole 50mg bid
Other Name: Placebo
Riluzole given as add-on therapy 50mg bid
Other Name: Rilutek
- Overall Survival Rate at 18 Months [ Time Frame: From the date of randomization until the date of death or last follow-up censored at 18 months (548 days) ]Overall survival was defined from the date of randomization until the date of death (event) or last known alive date (censored). If the death date was after 18 months, the participant was censored at 18 months (548 days). Participants still alive at or after 18 months were censored at 18 months/ 548 days. All data over the 18-month follow-up period after randomization, and participant survival status at the 18-month follow-up visit for participants who withdrew prematurely from the study for reasons other than death were included.
- Percentage of Participants With Failure Over 18 Months [ Time Frame: From randomization to the time of the first event to consider at 18 months (548 days) ]Time to failure was defined as the time from randomization to the time of the first event to consider (Tracheostomy, invasive ventilation [IV] or non invasive ventilation [NIV])
- Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: Inclusion, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 ]The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability).
- Percentage of Participants With a Global ALS FRS-R Score of <30 or Death [ Time Frame: Month 18 (548 days) ]Percentage of participants with a global ALS FRS-R score of < 30 or death was estimated using the Kaplan-Meier method in the ITT, with a two-tailed log-rank, both stratified by site of onset (bulbar or spinal) and non-stratified. The ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS participant's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability).
- Slow Vital Capacity (SVC) Percent Predicted [ Time Frame: Baseline, Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 ]SVC as a percent of the predicted value was evaluated and reported.
- Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months [ Time Frame: Month 18 (548 days) ]
- Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups [ Time Frame: Inclusion, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 ]MMT score involved the examination of 30 items. These 30 items are scored from 0 (no trace of contraction) to 5 (normal power at first try). The global score is the sum of the item scores and can range from 0 to 150. Higher score indicates some power.
- The Single-Item Mc Gill Quality of Life Scale [ Time Frame: Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18 ]The single-item McGill quality of life scale evaluated the following question "Considering all parts of my life - physical, emotional, social, spiritual, and financial - over the past two (2) days, the quality of my life has been…"as a score of 1 to 10 on a visual analog scale where 0 is very bad and 10 is excellent.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00868166
|University Hospital Gasthuisberg - Dept Neurology - Herestraat 49|
|Leuven, Belgium, 3000|
|HCL Hôpital Neurologique et Neurochirurgical Pierre Wertheimer - Neurologie C et Laboratoire d'électromyographie - 59, boulevard Pinel|
|Bron Cedex, France, 69677|
|CHRU de LILLE - Hôpital Roger Salengro - Centre SLA-MMN - Sce de Neurologie et Pathologie du Mouvement|
|Lille, France, 59037|
|Centre SLA Limoges - Service de Neurologie|
|Limoges, France, 87042|
|Hôpital La Timone - Service Neurologie et Maladies Neuromusculaires|
|Marseille, France, 13005|
|Clinique du Motoneurone - Sce d'Explorations Neurologiques - Hôpital Gui de Chauliac|
|Montpellier, France, 34295|
|CHU de Nice - Hôpital de l'Archet 1 - Centre de Référence pour les Maladies Neuromusculaires et la SLA|
|Nice, France, 06202|
|Groupe Hospitalier PITIE-SALPETRIERE - Fédération des Maladies du Système Nerveux|
|Paris, France, 75013|
|Charité Universitätsmedizin Berlin, Campus Virchow-Klinikum, Neurologische Poliklinik Ambulanz für ALS und andere Motoeneuronenerkrankungen|
|Berlin, Germany, 13353|
|Universitätsklinik und Poliklinik für Neurologie - Martin-Luther-Universität Halle-Wittenberg|
|Halle, Germany, 06097|
|Neurologische Klinik Medizinische Hochschule|
|Hannover, Germany, D-30623|
|Universitäts- und Rehabilitationskliniken Ulm (RKU) - Neurologische Universitätsklinik|
|Ulm, Germany, 89081|
|Hospital Carlos III - Unidad de ELA - Sinesio Delgado, 10|
|Madrid, Spain, 28029|
|King's MND Care and Research Center - Academic Neurosciences Building PO Box 41 Institute of Psychiatry|
|London, United Kingdom, SE58AF|
|Academic Neurology Unit - University of Sheffield - Section of Neuroscience - Division of Genomic Medicine - School of Medicine and Biomedical Sciences|
|Sheffield, United Kingdom, S10 2RX|
|Study Director:||Clinical Trials||Hoffmann-La Roche|