Clinical trial NCT04485481: Single and Multiple Ascending Dose Study of ADX-914 in Healthy Adult Volunteers
Official title: A Double-Blind, Phase 1, Single and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of ADX-914 in Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Safety Issues
Lead sponsor: Q32 Bio Inc.
Sponsor class: INDUSTRY
First posted: 2020-07-24
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: A two (2) part study to evaluate the safety, tolerability and PK of ADX-914

Detailed description: Part 1 - SAD:

It is expected that there will be up to 6 cohorts of 8 participants per cohort. In each cohort, participants will be randomly assigned in a 3:1 ratio to receive either ADX-914 or matching placebo.

It is planned that for each cohort in Part 1 a staggered 'sentinel' dose design will be used.

Part 2 - MAD It is expected that there will be up to 3 cohorts of 8 participants per cohort. In each cohort, participants will be randomly assigned in a 3:1 ratio to receive either ADX-914 or matching placebo. Doses will occur every 2 weeks, for a total of 4 doses.

Eligibility criteria: Inclusion Criteria:

* Healthy as determined by the Investigator, based on a medical evaluation including medical history, physical examination, laboratory tests and ECG recording
* Men and women age 18-50

Exclusion Criteria:

* Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels greater than 1.5 x the upper limit of normal (ULN) at Screening or Day -1.
* QT-interval measurements corrected according to the Fridericia rule (QTcF \>450 msec) during controlled rest at Screening or family history of long QT syndrome.
* Any clinically significant abnormalities in rhythm, conduction, or morphology of the resting ECG and any abnormalities in the 12-lead ECG that, in the judgment of the Investigator, may interfere with the interpretation of QTc-interval changes, including abnormal ST-T-wave morphology or left ventricular hypertrophy.
* A clinically significant vital signs abnormality, as judged by the Investigator, at Screening, or Day -1. This includes, but is not limited to, the following, in the supine position: (a) systolic blood pressure \<90 or \>140 mmHg, (b) diastolic blood pressure \<40 or \>90 mmHg, or (c) heart rate \<40 or \>100 beats per minute.

Primary outcomes:
measure: Number of subjects with adverse events
timeFrame: Screening to end of study, up to 18 weeks
measure: Number of subjects with Physical exam findings
timeFrame: Screening to end of study, up to 18 weeks
measure: Number of subjects with Clinical safety lab changes
timeFrame: Screening to end of study, up to 18 weeks
measure: Number of subjects with Systolic blood pressure changes
timeFrame: Screening to end of study, up to 18 weeks
measure: Number of subjects with Heart rate changes
timeFrame: Screening to end of study, up to 18 weeks
measure: Number of subjects with 12 Lead ECG changes
timeFrame: Screening to end of study, up to 18 weeks
Secondary outcomes:
measure: Maximum observed plasma concentration, Cmax
timeFrame: Predose to Day 91 (SAD) and Day 127 (MAD)
measure: Time to reach maximum observed plasma concentration, Tmax
timeFrame: Predose to Day 91 (SAD) and Day 127 (MAD)
measure: Area Under the plasma concentration time curve, AUC
timeFrame: Predose to Day 91 (SAD) and Day 127 (MAD)
Other outcomes:
Locations:
city: Melbourne
country: Australia
facility: Nucleus Network
state: Victoria
Study officials:
References: