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

• ClinicalTrials.gov Identifier: NCT04485481
• Recruitment Status: Recruiting
• First Posted: July 24, 2020
• Last Update Posted: September 11, 2020
• Sponsor: Q32 Bio Inc.
• Information provided by (Responsible Party): Q32 Bio Inc.

Tracking Information

• First Submitted Date: July 21, 2020
• First Posted Date: July 24, 2020
• Last Update Posted Date: September 11, 2020
• Actual Study Start Date: September 8, 2020
• Estimated Primary Completion Date: November 1, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 21, 2020)

• Number of subjects with adverse events [ Time Frame: Screening to end of study, up to 18 weeks ]
  Listing and summary of AE incidence
• Number of subjects with Physical exam findings [ Time Frame: 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 [ Time Frame: Screening to end of study, up to 18 weeks ]
  Listing and change from baseline to end of study
• Number of subjects with Systolic blood pressure changes [ Time Frame: Screening to end of study, up to 18 weeks ]
  Listing and change from baseline to end of study
• Number of subjects with Heart rate changes [ Time Frame: Screening to end of study, up to 18 weeks ]
  Listing and change from baseline to end of study
• Number of subjects with 12 Lead ECG changes [ Time Frame: Screening to end of study, up to 18 weeks ]
  Change in 12-lead ECG parameters from baseline to end of study

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 21, 2020)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Other
• Condition: Safety Issues

Intervention

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

Study Arms

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: July 21, 2020): 72
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: November 1, 2021
• Estimated Primary Completion Date: November 1, 2021 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 50 Years (Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: PI; 1800 243 733; recruitment@nucleusnetwork.com.au

• Listed Location Countries: Australia

Administrative Information

• NCT Number: NCT04485481
• Other Study ID Numbers: ADX-914-001
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Q32 Bio Inc.
• Study Sponsor: Q32 Bio Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Q32 Bio Inc.
• Verification Date: September 2020