A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

Launched by QURALIS CORPORATION · Nov 21, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called QRL-201 for people living with Amyotrophic Lateral Sclerosis (ALS), a condition that affects the nerves and muscles. The main goal of the study is to see how safe this treatment is and how well people can tolerate it when given in multiple doses. The trial is currently looking for participants aged 18 to 80 who have been diagnosed with ALS within the last two years and have some level of breathing ability.

To join the trial, participants must be able to undergo a procedure called a lumbar puncture, which involves taking a small sample of spinal fluid. They also need to be stable on any current ALS medications. However, certain individuals, such as those with specific genetic markers or other neurological conditions, will not be eligible to participate. If you or a family member are interested in learning more about this trial or if you might qualify, it could be a valuable opportunity to contribute to ALS research and receive close monitoring during the study.

Gender

ALL

Eligibility criteria

• 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 or electrodiagnostic 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
• • Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings.
• • An EEG that shows signs of abnormal electrical activity (e.g., epilepsy)