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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Healthy Volunteers

Intervention / Treatment
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* Drug: QRL-101
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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INCLUSION CRITERIA Age 18 to 70 years of age inclusive at the time of signing the informed consent. Clinical chemistry laboratory values within acceptable range for the population, as per investigator judgment. Body mass index of 18 to 32 kg/m2 (inclusive). Willing and able to practice effective contraception. EXCLUSION CRITERIA 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. Any participant in >4 studies a year and/or has participated in a clinical trial within 1 month of expected dosing date. 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*

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: This study will be a randomized, double-blind, placebo-controlled single ascending dose (SAD) study in healthy participants with the primary goal of evaluating the safety and tolerability of QRL-101.
Masking: Double


Arms and Interventions

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

What is the study measuring?
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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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Healthy Volunteers