A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis

Study Purpose:

The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS

Study Status:



Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

QRL-201 - Dose 1, QRL-201 - Dose 2, QRL-201 - Dose 3, QRL-201 - Dose 4, QRL-201 - Dose 5, QRL-201 - Dose 6, QRL-201 - Dose 7, QRL-201- Dose 8




Phase 1

Study Chair(s)/Principal Investigator(s):

Angela Genge, MD, QurAlis Corporation

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

QurAlis Corporation / email hidden; JavaScript is required / 617-720-9566

Full Study Summary:

This first-in-human, Phase 1 study will evaluate safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. 8 cohorts of 8 participants each, in a 6:2 ratio of QRL-201 to placebo will be tested.

Study Sponsor:

QurAlis Corporation

Estimated Enrollment:


Estimated Study Start Date:

12 / 16 / 2022

Estimated Study Completion Date:

05 / 06 / 2025

Posting Last Modified Date:

04 / 20 / 2023

Date Study Added to neals.org:

12 / 01 / 2022

Minimum Age:

18 Years

Maximum Age:

80 Years

Inclusion Criteria:

- Male or female participants aged 18 to 80 years diagnosed with ALS

- 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)

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

University of Calgary | Recruiting

/ 403-210-7009 / email hidden; JavaScript is required

Principal Investigator : Thomas Mobach, MD

Calgary, Alberta T2N 1N4

CHUM - Hopital Notre-Dame | Recruiting

/ 514-890-8000 Ext. 30737 / email hidden; JavaScript is required

Principal Investigator : Genevieve Matte, MD

Montréal, Quebec H2L 4M1

Montreal Neurological Institute-Hospital | Recruiting

/ 514-398-6183 / email hidden; JavaScript is required

Principal Investigator : Rami Massie, MD

Montréal, Quebec H3A 2B4