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

Phase 1

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: QRL-101; Other: Placebo

• Registration Number: NCT06714396
• Lead Sponsor: QurAlis Corporation

Brief 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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Study Timeline

• Status Verified: 2024-11
• Study Start: 2024-11-11
• Study First Submitted: 2024-11-27
• Study First Submitted QC: 2024-11-27
• Last Update Submitted: 2024-11-27
• Study First Posted: 2024-12-03
• Last Update Posted: 2024-12-03
• Primary Completion: 2025-03-10
• Study Completion: 2025-11-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
QRL-101	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.
Placebo	Placebo	Single doses of comparator placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.

Primary Outcome Measures

Name	Time	Method
QRL-101 plasma concentration	Baseline through Follow up (Day 10)
PK/PD Relationship: Strength-Duration Time Constant (SDTC)	Baseline through Follow up (Day 10)

Secondary Outcome Measures

Name	Time	Method
Safety and Tolerability: Adverse Events (AEs)	Baseline through Follow up (Day 10)
Safety and Tolerability: Serious Adverse Events (SAEs)	Baseline through Follow up (Day 10)
Plasma PK profile: Maximum concentration observed (Cmax)	Baseline through Follow up (Day 10)
Plasma PK profile: Time of maximum drug concentration (Tmax)	Baseline through Follow up (Day 10)
Plasma PK profile: Area under the concentration time curve from time 0 to 24 hours (AUC0-24hr)	Baseline through Follow up (Day 10)

Trial Locations

Locations (1)

Universitair Medisch Centrum Utrecht; 🇳🇱 Utrecht, Netherlands