FUSION: a multicentre, double-blind randomised placebo-controlled phase I-III study to investigate the effect, safety, pharmacokinetics and pharmacodynamics of intrathecally administered ION363 in patients with amyotrophic lateral sclerosis (ALS) with an FUS mutation.

This phase I-III study is sponsored by IONIS Pharmaceuticals and aims to determine whether reducing FUS protein levels using antisense oligonucleotides directed against the messenger RNA molecule of FUS has a beneficial effect in ALS patients with an FUS mutation.

FUS mutations are a rare inherited cause of ALS and precipitation of the protein FUS occurs in these patients. ION363 is an antisense oligonucleotide or ASO administered intrathecally to inhibit the formation of the FUS protein. A dose of 100 mg is being investigated in this study; after several loading doses, the drug will be administered once every three months. In the first phase, 1 in 3 patients will receive placebo. A second phase of the study is an ‘open label’ study, in which all patients will be administered the active product. Patients who deteriorate significantly during the first part of the study will be able to switch to the second part of the study at an accelerated rate. Globally, 50-60 patients will be included.

Interested, or would you like further information? Contact us at onderzoek@als.be

More info on this study can be found on the website:
https://clinicaltrials.gov/ct2/show/NCT04768972?term=ion363&cond=ALS&draw=2&rank=1