ANQUR
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
UK Chief Investigator
Professor Chris McDermott
Research summary
This is a first-in-human, randomised, double-blind study. This study will evaluate multiple dose levels of QRL-201, and participants will be enrolled into the study in small groups of approximately 8 participants each, called cohorts.
This study will include approximately 64 participants. The dose level of QRL-201 will increase with each group for Cohorts 1 through 6. Cohorts 7 through 8 will have the same dose level. Study drug will be administered up to 5 times to each participant over a period of approximately 3 months.
The study duration will be approximately 40 weeks: 4 weeks of screening, 12 weeks for study drug administration, 20 weeks for terminal PK collection and a final follow-up visit at 36 weeks after the first study drug administration.
The planned study duration is 30 months from screening of the first participant until the last follow-up of the last participant.
This trial is placebo-controlled. It is important to understand what this means. Click the links to go to the glossary of terms and read further explanations.
- What the chances are of you being randomly assigned to either an intervention group or a placebo group
- How long you would remain on this treatment allocation
- If the study offers an open-label extension on completion of the main study.
Current status
In set-up, opening soon
Recruitment target
8 participants in the UK; up to 64 participants globally
Recruitment group(s)
Patients with MND
Locations
Sheffield (Principal Investigator: Chris McDermott)
King's College Hospital (Principal Investigator: Ammar Al-Chalabi)
Contact details
Email: clinicaltrials@quralis.com
Key dates
Planned recruitment opening date: April 2023
Planned recruitment end date: 30 November 2024
Inclusion / exclusion criteria
Key inclusion criteria:
Male or female participants aged 18 to 80 years diagnosed with ALS
ALS symptom onset within 24 months of Screening
Slow vital capacity >50%
Clinical evidence of lower motor neuron involvement
Not pregnant and not nursing
Willing and able to practice effective contraception
Able to tolerate lumbar puncture
If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion)
Key exclusion criteria:
Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes
Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device
Prior exposure to stem cell or gene therapy products
Any contraindication to intrathecal drug administration
Abnormal laboratory values deemed clinically significant by the Investigator
Significant infection, or known inflammatory process
Funder(s)
QurAlis Corporation
Sponsor
QurAlis Corporation
Study design
Interventional, randomised controlled trial
Intervention
Drug: QRL-201
Phase
Outcome measures
Primary outcome measure
Number of participants with one or more treatment emergent adverse events and serious adverse events
Secondary outcome measures
Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)
Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201
Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201