NCT06714396

A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS

A Randomized, Placebo-Controlled, Double-Blind, Single-Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: QurAlis Corporation · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.

Detailed description

This is a randomized, placebo-controlled, double-blind, single-dose, single-site, Phase 1 study to explore the pharmacokinetics (PK) and pharmacodynamics (PD) of single doses of QRL-101 in participants with ALS. In addition, the safety and tolerability of QRL-101 will be evaluated. Single doses of QRL-101 or placebo will be administered as an oral liquid to approximately 12 participants with ALS. The study will explore the PK, PD, and PK/PD relationship among three dose levels of QRL-101 and placebo. The randomization will be 1:1:1:1 for the four study groups and will be conducted in 3 blocks (cohorts) of 4, where participants will be randomized 3:1 (QRL-101:placebo) for increasing dose levels of QRL-101.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	QRL-101	Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
OTHER	Placebo	Single doses of compartor placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

Timeline

• Start date: 2024-11-11
• Primary completion: 2025-08-22
• Completion: 2025-08-22
• First posted: 2024-12-03
• Last updated: 2025-09-02

Locations

1 site across 1 country: Netherlands