Single and Multiple Ascending Dose Study of ADX-914 in Healthy Adult Volunteers

Phase 1

Completed

• Conditions: Safety Issues
• Interventions: Drug: ADX-914; Drug: Placebo

• Registration Number: NCT04485481
• Lead Sponsor: Q32 Bio Inc.

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.

Study Timeline

• Study First Submitted: 2020-07-21
• Study First Submitted QC: 2020-07-21
• Study First Posted: 2020-07-24
• Study Start: 2020-09-08
• Primary Completion: 2022-01-12
• Study Completion: 2022-01-12
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-27
• Last Update Posted: 2022-07-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 42

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Experimental: Cohort 1:1 - 1:6 ADX-914	ADX-914	ADX-914 single SC dose
Placebo Comparator: Cohort 1:1 - 1:6 placebo	Placebo	Placebo single SC dose
Experimental: Cohort 2:1- 2:3	ADX-914	ADX-914 multiple SC dose once every 2 weeks for 6 weeks
Placebo Comparator: Cohort 2:1- 2:3	Placebo	Placebo multiple SC dose once every 2 weeks for 6 weeks

Primary Outcome Measures

Name	Time	Method
Number of subjects with 12 Lead ECG changes	Screening to end of study, up to 18 weeks	Change in 12-lead ECG parameters from baseline to end of study
Number of subjects with Heart rate changes	Screening to end of study, up to 18 weeks	Listing and change from baseline to end of study
Number of subjects with Physical exam findings	Screening to end of study, up to 18 weeks	Listing of clinically significant changes in PE findings
Number of subjects with Clinical safety lab changes	Screening to end of study, up to 18 weeks	Listing and change from baseline to end of study
Number of subjects with adverse events	Screening to end of study, up to 18 weeks	Listing and summary of AE incidence
Number of subjects with Systolic blood pressure changes	Screening to end of study, up to 18 weeks	Listing and change from baseline to end of study

Secondary Outcome Measures

Name	Time	Method
Maximum observed plasma concentration, Cmax	Predose to Day 91 (SAD) and Day 127 (MAD)	Of ADX-914
Time to reach maximum observed plasma concentration, Tmax	Predose to Day 91 (SAD) and Day 127 (MAD)	Of ADX-914
Area Under the plasma concentration time curve, AUC	Predose to Day 91 (SAD) and Day 127 (MAD)	Of ADX-914

Trial Locations

Locations (1)

Nucleus Network; 🇦🇺 Melbourne, Victoria, Australia