NCT05633459

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

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

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 64 (estimated)
• Sponsor: QurAlis Corporation · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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

Detailed description

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Multiple ascending doses of QRL-201	Multiple ascending doses of QRL-201 will be intrathecally administered to individuals with sporadic ALS.
DRUG	Multiple ascending doses of Placebo	Multiple ascending doses of placebo comparator will be intrathecally administered to individuals with sporadic ALS.
DRUG	QRL-201	QRL-201 will be intrathecally administered to individuals with C9orf72 ALS.
DRUG	Placebo	Placebo comparator will be intrathecally administered to individuals with C9orf72 ALS.

Timeline

• Start date: 2022-12-16
• Primary completion: 2026-10-08
• Completion: 2026-10-08
• First posted: 2022-12-01
• Last updated: 2025-08-29

Locations

15 sites across 6 countries: Belgium, Canada, Germany, Ireland, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study