What does the drug do?
QRL-201 aims to restore STATHMIN-2 expression in people living with MND. STATHMIN-2 is a protein that is important for neurone repair and stability of axons, which carry the electrical impulse from the cell body to the end of the neurone. In almost all people living with ALS, STATHMIN-2 expression is significantly decreased. In pre-clinical studies QRL-201 has been found to increase the expression of STATHMIN-2, including in human models of ALS (using induced pluripotent stem cells (iPSCs)).
Phase 1 - ANQUR
Current status: In preparation
ANQUR is the first in-human, randomised, double-blind study which is designed to evaluate the safety and tolerability of QRL-201 in people living with ALS. The study will also evaluate multiple doses of QRL-201, with participants being placed into small groups when they enrolled into the study. Participants will be given the drug for 12 weeks but additional information will be taken up to 36 weeks after first receiving the drug. Find out more on the MND Clinical Studies Group website or on a clinicaltrials.gov.
How can I take part in the trial?
Recruiting Sites
The trial is currently in set up in the UK. We will update this page with trial sites once the trial has begun recruiting.
Trial Inform
Phase: Phase 1
Placebo Controlled: Yes - this means that people joining the trial could be randomly assigned to receive the placebo (dummy drug).
Open-Label Extension: No - this means that participants will only receive either the treatment or the placebo throughout the trial.
Key eligibilty criteria: Participants cannot enter this trial if they have mutations in the SOD1 or FUS genes. More criteria here.
Latest News
2023
Jan 2023 - The trial began recruiting in Canada.
Last updated: 11/03/2024