Amyotrophic lateral sclerosis (ALS) research is advancing quickly, but the transition from phase II to phase III trials remains particularly challenging. In part, this is because of the paradox of phase II ALS trials - they are expected to inform researchers about safety, tolerability, dosage selection, and efficacy using a small number of patients, and relying on essentially the same outcome measures used in phase III trials. We examined pharmacokinetics in the cerebrospinal fluid and pharmacodynamic markers to demonstrate target engagement. In addition, primary outcome measures are often not specified or do not reach pre-specified goals for significance. We conclude that future trials should include pharmacokinetic (preferably in CSF) and pharmacodynamic markers of target engagement when possible. Primary endpoints should be pre-specified. Inclusion criteria should be used to reduce heterogeneity and target a relevant subpopulation of people with ALS when possible. Multiple phase II trials might be required before moving to a large phase III trial.
This research output has an Altmetric Attention Score of 4. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 11 March 2025.
All research outputs
#8,079,169
of 27,246,312 outputs
Outputs from Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
#696
of 1,201 outputs
Outputs of similar age
#69,638
of 239,836 outputs
Outputs of similar age from Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
#4
of 13 outputs
Altmetric has tracked 27,246,312 research outputs across all sources so far. This one has received more attention than most of these and is in the 69th percentile.
So far Altmetric has tracked 1,201 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 10.3. This one is in the 41st percentile – i.e., 41% of its peers scored the same or lower than it.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 239,836 tracked outputs that were published within six weeks on either side of this one in any source. This one has gotten more attention than average, scoring higher than 69% of its contemporaries.
We're also able to compare this research output to 13 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 69% of its contemporaries.
