A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants

A Randomized, Placebo-Controlled, Double-blind, Single Ascending Dose, First in Human Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending single doses of QRL-101 in male and female healthy participants. The findings from this study will be used to inform the development of QRL-101 for people living with ALS.

Study Overview

• Status: Completed
• Conditions: Healthy Volunteers
• Intervention / Treatment: Drug: QRL-101; Other: Placebo

Detailed Description

Phase 1, single-site study to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending single doses of QRL-101 administered orally in healthy male and female participants. Up to 16 cohorts of 8 participants each, randomized 6:2 (QRL-101: placebo) will be tested. The approximate total duration of study participation for each participant may be up to 40 days.

• Study Type: Interventional
• Enrollment (Actual): 88
• Phase: Phase 1

Contacts and Locations

Study Locations

• Netherlands
  • Groningen, Netherlands, 9728 NZ
    • ICON plc. Van Swietenlaan 6

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

INCLUSION CRITERIA

1. Age 18 to 70 years of age inclusive at the time of signing the informed consent.
2. Clinical chemistry laboratory values within acceptable range for the population, as per investigator judgment.
3. Body mass index of 18 to 32 kg/m2 (inclusive).
4. Willing and able to practice effective contraception.

EXCLUSION CRITERIA

1. Currently enrolled in any other clinical trial involving a study drug or off-label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study.
2. Any participant in >4 studies a year and/or has participated in a clinical trial within 1 month of expected dosing date.
3. History or presence of medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality that, in the judgment of the investigator, indicate a medical problem that would preclude study participation.

*Other inclusion and exclusion criteria may apply*

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: QRL-101; Single-ascending doses of QRL-101 will be administered orally to healthy participants	Drug: QRL-101; Single-ascending doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
Placebo Comparator: Placebo; Single-ascending doses of comparator placebo will be administered orally to healthy participants	Other: Placebo; A placebo comparator will be administered at all dose levels.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with one or more treatment emergent adverse events and serious adverse events.	Endpoints: A summary of treatment emergent adverse events (AEs), serious adverse events (SAEs), and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module.	Baseline through Follow up (Day 10)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics (plasma): Maximum observed concentration of QRL-101	Endpoint: Maximum observed concentration (Cmax) of QRL-101	Baseline through Follow up (Day 10)
Pharmacokinetics (plasma): Area under the concentration time curve from 0 to 24 h (AUC 0-24h) of QRL-101	Endpoint: Area under the concentration time curve from zero to infinity (AUC 0-24h) of QRL-101	Baseline through Follow up (Day 10)
Pharmacokinetics (plasma): Time of maximum concentration of QRL-101	Endpoint: Time of maximum concentration (Tmax) of QRL-101	Baseline through Follow up (Day 10)

Collaborators and Investigators

• Sponsor: QurAlis Corporation
• Investigators: Principal Investigator: Salah Hadi, MD, ICON plc

Study record dates

Study Major Dates

• Study Start (Actual): December 22, 2022
• Primary Completion (Actual): December 27, 2023
• Study Completion (Actual): December 27, 2023

Study Registration Dates

• First Submitted: December 19, 2022
• First Submitted That Met QC Criteria: December 19, 2022
• First Posted (Actual): December 29, 2022

Study Record Updates

• Last Update Posted (Estimated): April 15, 2024
• Last Update Submitted That Met QC Criteria: April 12, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Healthy Volunteers
• Other Study ID Numbers: QRL-101-01; 2022-002484-30 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes