RANDOMISED DOUBLE-BLIND PLACEBO-CONTROLLED PHASE 3 TRIAL OF TRIUMEQ IN AMYOTROPHIC LATERAL SCLEROSIS - LIGHTHOUSE II

Conditions

AMYOTROPHIC LATERAL SCLEROSIS

Brief summary

The primary endpoint is overall survival, defined as time to mortality from any cause

Detailed description

Combined assessment of survival and measures of daily functioning using the ALSFRS-R(30) total score (CAFS), Daily functioning using the ALSFRS-R(30) total score, Respiratory function measured by vital capacity (VC) (% predicted of normal according to the GLI-2012 reference standard(37)), Plasma creatinine levels, Clinical disease stage, defined as mean time spent in each stage of the King’s Staging Scale and the ALS Milano-Torino staging systems (36) (MITOS, derived from ALSFRS-R(30)), Safety based on the safety assessments including physical examinations, clinical laboratory evaluations, vital signs and frequency of adverse events (AEs) or serious adverse events (SAEs). (S)A Es will be categorized according seriousness, causality (by study medication), severity and expectedness (as defined in the Triumeq Summary of Product Characteristics), Tolerabilty, as defined by study medication discontinuation, Cognitive function, defined as the total scores on the ECAS(32), Quality of life, defined as total scores on the Visual Analogue Scale (single-item scale) and EQ-5D-5L(35), Laboratory parameters e.g. Urine P75ECD, plasma neurofilament light and heavy chain, HERV-K expression and genotyping (UNC13a / C9orf72)

Related papers

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

Interventions

DRUGThe placebo capsules have a formulation that matches crushed Triumeq tablets regarding appearance and taste.

DRUGTriumeq 50 mg/600 mg/300 mg film-coated tablets

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The primary endpoint is overall survival, defined as time to mortality from any cause	—

Secondary

Measure	Time frame
Combined assessment of survival and measures of daily functioning using the ALSFRS-R(30) total score (CAFS), Daily functioning using the ALSFRS-R(30) total score, Respiratory function measured by vital capacity (VC) (% predicted of normal according to the GLI-2012 reference standard(37)), Plasma creatinine levels, Clinical disease stage, defined as mean time spent in each stage of the King’s Staging Scale and the ALS Milano-Torino staging systems (36) (MITOS, derived from ALSFRS-R(30)), Safety based on the safety assessments including physical examinations, clinical laboratory evaluations, vital signs and frequency of adverse events (AEs) or serious adverse events (SAEs). (S)A Es will be categorized according seriousness, causality (by study medication), severity and expectedness (as defined in the Triumeq Summary of Product Characteristics), Tolerabilty, as defined by study medication discontinuation, Cognitive function, defined as the total scores on the ECAS(32), Quality of life, de	—

Measure

Time frame

Combined assessment of survival and measures of daily functioning using the ALSFRS-R(30) total score (CAFS), Daily functioning using the ALSFRS-R(30) total score, Respiratory function measured by vital capacity (VC) (% predicted of normal according to the GLI-2012 reference standard(37)), Plasma creatinine levels, Clinical disease stage, defined as mean time spent in each stage of the King’s Staging Scale and the ALS Milano-Torino staging systems (36) (MITOS, derived from ALSFRS-R(30)), Safety based on the safety assessments including physical examinations, clinical laboratory evaluations, vital signs and frequency of adverse events (AEs) or serious adverse events (SAEs). (S)A Es will be categorized according seriousness, causality (by study medication), severity and expectedness (as defined in the Triumeq Summary of Product Characteristics), Tolerabilty, as defined by study medication discontinuation, Cognitive function, defined as the total scores on the ECAS(32), Quality of life, de

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Countries

Ireland, Netherlands, Slovenia, Spain, Sweden

Outcome results

None listed