Transposon studie with TPN-101 in C9orf72 ALS and FTD
A phase 2a multicenter, randomized, double blind , placebo controlled study of TPN-101 in patients with C9ORF72 ALS (amyotrophic lateral sclerosis) and/or FTD( frontotemporal dementia) with subsequent open label treatment phase. The study includes a 6-week screening period followed by a 24-week double-blind phase in which 3:2 patients will be administered TPN101. Then there is an open label phase in which everyone will receive the active product for 24 weeks.
This phase 2a study is sponsored by the company Transposon. In ALS and FTD, it seems that cells lose their ability to prevent junk DNA from becoming active. Junk DNA can affect cells and lead to inflammation and damage to functional DNA. As a result, it may play a role in the death of motor neurons in C9orf72 ALS. The goal of the oral study drug TPN -101 is just to block the effects of junk DNA.
Approximately 40 patients worldwide with a C9orf72 gene and a diagnosis of ALS and/or FTD will be involved in this study.
More info about this study can be found on the website:
https://www.clinicaltrials.gov/ct2/show/NCT04993755?cond=amyotrophic+C9o...
Interested, or desire further information? Contact us at onderzoek@als.be
