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.

Current status

In set-up, opening soon

Recruitment target

8 participants in the UK; up to 64 participants globally 

Recruitment groups

Patients with Amyotrophic Lateral Sclerosis (ALS), a type of MND

Locations

• London - King's College Hospital, Principal Investigator: Ammar Al-Chalabi.  Site open for recruitment out-of-area

• Sheffield, Principal Investigator: Chris McDermott. Site open for local recruitment only

Information about study sites

Contact details

Email: clinicaltrials@quralis.com

Key dates

Recruitment opening date: 31 October 2024

Planned recruitment end date: 30 May 2025

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 

• Placebo

Phase

1

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