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

Safety Issues Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04485481
• Other study ID #: ADX-914-001
• Status: Completed
• Phase: Phase 1
• Start date: September 8, 2020
• Est. completion date: January 12, 2022

Study information

• Verified date: July 2022
• Source: Q32 Bio Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 42
• Est. completion date: January 12, 2022
• Est. primary completion date: January 12, 2022
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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.

Related Conditions & MeSH terms

• Safety Issues

Intervention

Drug:
• ADX-914
Single dose from 0.1mg/kg to TBD
• Placebo
Matching single dose placebo
• ADX-914
Multiple dose from TBD to TBD
• Placebo
Matching multiple dose placebo

Locations

Country	Name	City	State
Australia	Nucleus Network	Melbourne	Victoria

Sponsors (1)

Lead Sponsor	Collaborator
Q32 Bio Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with adverse events	Listing and summary of AE incidence	Screening to end of study, up to 18 weeks
Primary	Number of subjects with Physical exam findings	Listing of clinically significant changes in PE findings	Screening to end of study, up to 18 weeks
Primary	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
Primary	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
Primary	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
Primary	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	Maximum observed plasma concentration, Cmax	Of ADX-914	Predose to Day 91 (SAD) and Day 127 (MAD)
Secondary	Time to reach maximum observed plasma concentration, Tmax	Of ADX-914	Predose to Day 91 (SAD) and Day 127 (MAD)
Secondary	Area Under the plasma concentration time curve, AUC	Of ADX-914	Predose to Day 91 (SAD) and Day 127 (MAD)