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

• Status: Not yet recruiting
• Trial end date: 2025-05-06
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: QurAlis Corporation

Criteria

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