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Platform Trial to Assess the Efficacy of Multiple Drugs in Amyotrophic Lateral Sclerosis (ALS)
The objective of this phase III, placebo-controlled platform study is to investigate the efficacy of drugs for patients with ALS (Amyotrophic lateral sclerosis).
Study details:
This study uses an innovative multi-arm, adaptive trial design to investigate the efficacy of multiple treatments simultaneously. Currently one study-arm is active, investigating the efficacy and safety of lithium carbonate versus placebo in patients with ALS. Only patients with a specific UNC13A genotype (approximately 1 in 6 ALS patients) are eligible to participate.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 18 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2021-08-09
Primary completion: 2026-06-01
Study completion finish: 2026-06-01
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT06008249
Intervention or treatment
DRUG: Lithium Carbonate 400 MG
Conditions
- • Amyotrophic Lateral Sclerosis
Find a site
Closest Location:
Flinders Medical Centre
Research sites nearby
Select from list below to view details:
Flinders Medical Centre
Adelaide, Not Specified, Australia
Royal Brisbane and Women's Hospital
Brisbane, Not Specified, Australia
Calvary Health Care Bethlehem
Parkdale, Not Specified, Australia
Perron Institute
Perth, Not Specified, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Lithium carbonate
| DRUG: Lithium Carbonate 400 MG
|
PLACEBO_COMPARATOR: Placebo
| DRUG: Lithium Carbonate 400 MG
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Overall survival, defined as time to death from any cause or respiratory insufficiency (DRI; defined as tracheostomy or the use of non-invasive ventilation for ≥22 h per day for ≥10 consecutive days) | A tracheostomy for ventilation is meant here | endpoint or 24 months |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Composite endpoint evaluating daily functioning and survival based on the joint model framework of survival and longitudinal ALSFRS-R total scores | The ALSFRS-R (Amyotrophic Lateral Sclerosis Rating Scale-revised) is a 12 item participant self-report measure that monitors ALS disease progression, where a higher score reflects a better outcome. | endpoint or 24 months |
Daily functioning, defined as mean change from baseline in ALSFRS-R total score. | The ALSFRS-R (Amyotrophic Lateral Sclerosis Rating Scale-revised) is a 12 item participant self-report measure that monitors ALS disease progression, where a higher score reflects a better outcome. | endpoint or 24 months |
Respiratory function, defined as mean change from baseline in SVC (%predicted of normal according to the GLI-2012 reference standard) | Slow vital capacity (SVC) is measured in litres, and as a % of predicted. A higher score reflects a better outcome. | endpoint or 24 months |
Quality of life, defined as change from baseline on the EQ-5D Visual Analogue Scale (single-item scale) | The EQ-5D-5L (EuroQol 5 Dimension 5 Level) questionnaire is a standardised measure of health-related Quality of Life, using a Visual Analogue Scale. A higher score relates to a better outcome | endpoint or 24 months |
Quality of life, defined as change from baseline on the EQ-5D | The EQ-5D-5L (EuroQol 5 Dimension 5 Level) questionnaire is a standardised measure of health-related Quality of Life. A lower score relates to a better outcome | endpoint or 24 months |
Neuropsychological status, defined as change from baseline on the ECAS | ECAS (Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen) is a multidomain assessment questionnaire used in ALS to assess cognitive and behavioural changes where a higher score relates to a better outcome. | endpoint or 24 months |
Neuropsychological status, defined as change from baseline on the ALS-FTD-Q. | ALS-FTD-Q (Amyotrophic Lateral Sclerosis-Frontotemporal Dementia-Questionnaire) is a validated instrument for the screening of behavioral disturbances in ALS. | endpoint or 24 months |
Clinical disease stage, defined as mean time spent in each stage of the King's and ALS Milano-Torino staging systems. | The King's Staging Scale is a clinical staging system defining four stages of ALS assessed by way of a semi-structured interview with the participant. | endpoint or 24 months |
Change from baseline in laboratory parameters: Urinary P75ECD (ectodomain of neurotrophin receptor p75), Neurofilament light and heavy chain, Plasma creatinine | Plasma creatinine is assessed to monitor kidney function | endpoint or 24 months |
Tolerability defined as time-to-discontinuation of assigned treatment since randomization | the number of participants who discontinue study medication will be assessed to assess tolerability | endpoint or 24 months |
Safety based on the safety assessments including neurological examinations, clinical laboratory evaluations, vital signs and frequency of adverse events (AEs) or serious adverse events (SAEs). | (S)AEs will be categorized according to the Common Terminology Criteria for Adverse Events and will be rated for severity and association with study drug. | endpoint or 24 months |
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