The trial, conducted in America, suggested that NP001 is safe, well tolerated and could be beneficial for MND.
Following these encouraging results, Neuraltus plan to begin a larger, Phase III trial of NP001 in the second half of 2013. As the Phase III trial is still being planned, we do not have details on American recruitment centres, nor what the eligibility criteria will be.
The Phase II clinical trial for NP001 was rigorously controlled. This means that it was randomised, double-blinded and placebo controlled. These are important factors in controlling possible bias. We have more information on why these factors are important on our website.
The trial included 136 people living with MND in America across multiple centres.
Participants were randomised into three groups to receive an intravenous infusion of either high dose NP001, low dose NP001 or placebo (inactive substance) treatment for six months. They were then followed for an additional six months. Approximately 45 people were used in each treatment arm.
Results suggest that the treatment was safe and well tolerated. Promising signs of effectiveness were also identified, but were not statistically significant to draw firm conclusions as to whether the treatment could be effective for MND.
The trial organisers state in their press release that 27% of people taking the high-dose NP001 did not progress during the trial period. It’s important to treat these results with a certain degree of caution, as approximately 10% of people taking the inactive placebo also did not progress during the same period as measured by changes in the functional rating scale.
The results provide enough evidence to warrant a larger scale trial to investigate this treatment further.
This finding also importantly identifies the optimum dose that should be used in this larger-scale clinical trial, as their results suggest that a higher dose could be more likely to yield a beneficial effect than the lower dose.
Finding out the optimal dose is an important part of Phase II clinical trials to ‘fine tune’ the details to provide the treatment with the best chances of demonstrating its success at Phase III.
Importance of sharing results via peer-review
These results will need to be published in a peer-reviewed scientific journal. Peer review is an important process to determine whether findings are valid and that appropriate standards have been used in the study. Once published, these findings will also be used by the scientific community to add to their knowledge.
Importance of Phase III planned for 2013
The promising results identified in Phase II will need to be confirmed in the Phase III trial planned for the second half of 2013.
Leading UK clinical trial researcher, Prof Nigel Leigh said, “A larger Phase III randomised placebo-controlled trial is required before we can be confident that these positive trends are consistent and clinically significant.”
Dr Brian Dickie, the MND Association’s Director of Research continues, “We welcome Neuraltus’ plan to initiate a Phase III trial to determine whether NP001 is beneficial for people living with MND.”
Discussing results at the International Symposium on ALS/MND
Results from the Phase II NP001 trial will be discussed in more detail at the 23rd International Symposium on ALS/MND, to be held on 5-7 December 2012.
The symposium, organised by the MND Association provides a platform for researchers, clinicians and healthcare professionals to discuss the latest developments in research and care, including discussing results from recent clinical drug trials.
We will be reporting live from the symposium via our research blog. To ensure you have daily updates from the symposium, please sign up to our automatic email alerts on the right hand side of the blog.