Clinical trial NCT05667779: A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy Volunteers
Keywords: Healthy Volunteers
Lead sponsor: QurAlis Corporation
Sponsor class: INDUSTRY
First posted: 2022-12-29
Enrollment: 88
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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\*

Primary outcomes:
measure: Number of participants with one or more treatment emergent adverse events and serious adverse events.
timeFrame: Baseline through Follow up (Day 10)
Secondary outcomes:
measure: Pharmacokinetics (plasma): Maximum observed concentration of QRL-101
timeFrame: Baseline through Follow up (Day 10)
measure: Pharmacokinetics (plasma): Area under the concentration time curve from 0 to 24 h (AUC 0-24h) of QRL-101
timeFrame: Baseline through Follow up (Day 10)
measure: Pharmacokinetics (plasma): Time of maximum concentration of QRL-101
timeFrame: Baseline through Follow up (Day 10)
Other outcomes:
Locations:
city: Groningen
country: Netherlands
facility: ICON plc. Van Swietenlaan 6
Study officials:
affiliation: ICON plc
name: Salah Hadi, MD
role: PRINCIPAL_INVESTIGATOR
References: