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

A Double-Blind, Phase 1, Single and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of ADX-914 in Healthy Volunteers

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

Study Overview

• Status: Completed
• Conditions: Safety Issues
• Intervention / Treatment: Drug: ADX-914; Drug: ADX-914; Drug: Placebo; Drug: Placebo

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 Type: Interventional
• Enrollment (Actual): 42
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • Nucleus Network

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 48 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: Cohort 1:1 - 1:6 ADX-914; ADX-914 single SC dose	Drug: ADX-914; Single dose from 0.1mg/kg to TBD; Drug: ADX-914; Multiple dose from TBD to TBD
Placebo Comparator: Placebo Comparator: Cohort 1:1 - 1:6 placebo; Placebo single SC dose	Drug: Placebo; Matching single dose placebo; Drug: Placebo; Matching multiple dose placebo
Experimental: Experimental: Cohort 2:1- 2:3; ADX-914 multiple SC dose once every 2 weeks for 6 weeks	Drug: ADX-914; Single dose from 0.1mg/kg to TBD; Drug: ADX-914; Multiple dose from TBD to TBD
Placebo Comparator: Placebo Comparator: Cohort 2:1- 2:3; Placebo multiple SC dose once every 2 weeks for 6 weeks	Drug: Placebo; Matching single dose placebo; Drug: Placebo; Matching multiple dose placebo

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Q32 Bio Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 8, 2020
• Primary Completion (Actual): January 12, 2022
• Study Completion (Actual): January 12, 2022

Study Registration Dates

• First Submitted: July 21, 2020
• First Submitted That Met QC Criteria: July 21, 2020
• First Posted (Actual): July 24, 2020

Study Record Updates

• Last Update Posted (Actual): July 28, 2022
• Last Update Submitted That Met QC Criteria: July 27, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Other Study ID Numbers: ADX-914-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No