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

Conditions de participation

• Sexe:

  ALL

• Âges admissibles:

  18 to 80

Critères de participation

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)