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

Trial ID: NCT05633459

Interventional - Phase 1 - Active, not recruiting

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

Conditions

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)

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)

QurAlis Corporation