The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
This first-in-human, Phase 1 study will evaluate the safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. Two dose escalation cohorts of 8 participants each, followed by an additional 48 participants, receiving the study drug in a 6:2 ratio of QRL-201 to placebo.
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)