ISIS Pharmaceuticals Initiates a Phase IB/IIA Trial of ISIS-SMNRx in SMA Patients
ISIS Pharmaceuticals Initiates a Phase IB/IIA Trial of ISIS-SMNRx in SMA Patients
Original article published 7th November 2012
Isis Pharmaceuticals has started a Phase Ib/IIa clinical trial of ISIS-SMNRx in 24 children with SMA (ages 2-15). Following on from the Phase Ia safety trial completed earlier this year, this new study aims to assess the safety, tolerability, and pharmacokinetic profile of increasing doses of the drug.
ISIS-SMNRx, which is an antisense oligonucleotide drug able to increase the amount of available SMN protein in SMA model mice, will be delivered into thespinal cord of each patient two or three times over the course of the trial (36 weeks). Three different dose levels (8 patients per dose) will be assessed and the number of participants with adverse reactions recorded as the primary outcome measure. The amount of drug found in the blood plasma and cerebrospinal fluid will also be measured.
It is hoped that the data generated from this study will help to determine the dose that will be tested in later clinical trials in patients with SMA.