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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04485481




Registration number
NCT04485481
Ethics application status
Date submitted
21/07/2020
Date registered
24/07/2020
Date last updated
11/09/2020

Titles & IDs
Public title
Single and Multiple Ascending Dose Study of ADX-914 in Healthy Adult Volunteers
Scientific 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
Secondary ID [1] 0 0
ADX-914-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Safety Issues 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ADX-914
Treatment: Drugs - Placebo
Treatment: Drugs - ADX-914
Treatment: Drugs - Placebo

Experimental: Experimental: Cohort 1:1 - 1:6 ADX-914 - ADX-914 single SC dose

Placebo Comparator: Placebo Comparator: Cohort 1:1 - 1:6 placebo - Placebo single SC dose

Experimental: Experimental: Cohort 2:1- 2:3 - ADX-914 multiple SC dose once every 2 weeks for 6 weeks

Placebo Comparator: Placebo Comparator: Cohort 2:1- 2:3 - Placebo multiple SC dose once every 2 weeks for 6 weeks


Treatment: Drugs: ADX-914
Single dose from 0.1mg/kg to TBD

Treatment: Drugs: Placebo
Matching single dose placebo

Treatment: Drugs: ADX-914
Multiple dose from TBD to TBD

Treatment: Drugs: Placebo
Matching multiple dose placebo

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of subjects with adverse events - Listing and summary of AE incidence
Timepoint [1] 0 0
Screening to end of study, up to 18 weeks
Primary outcome [2] 0 0
Number of subjects with Physical exam findings - Listing of clinically significant changes in PE findings
Timepoint [2] 0 0
Screening to end of study, up to 18 weeks
Primary outcome [3] 0 0
Number of subjects with Clinical safety lab changes - Listing and change from baseline to end of study
Timepoint [3] 0 0
Screening to end of study, up to 18 weeks
Primary outcome [4] 0 0
Number of subjects with Systolic blood pressure changes - Listing and change from baseline to end of study
Timepoint [4] 0 0
Screening to end of study, up to 18 weeks
Primary outcome [5] 0 0
Number of subjects with Heart rate changes - Listing and change from baseline to end of study
Timepoint [5] 0 0
Screening to end of study, up to 18 weeks
Primary outcome [6] 0 0
Number of subjects with 12 Lead ECG changes - Change in 12-lead ECG parameters from baseline to end of study
Timepoint [6] 0 0
Screening to end of study, up to 18 weeks
Secondary outcome [1] 0 0
Maximum observed plasma concentration, Cmax - Of ADX-914
Timepoint [1] 0 0
Predose to Day 91 (SAD) and Day 127 (MAD)
Secondary outcome [2] 0 0
Time to reach maximum observed plasma concentration, Tmax - Of ADX-914
Timepoint [2] 0 0
Predose to Day 91 (SAD) and Day 127 (MAD)
Secondary outcome [3] 0 0
Area Under the plasma concentration time curve, AUC - Of ADX-914
Timepoint [3] 0 0
Predose to Day 91 (SAD) and Day 127 (MAD)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
50 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key 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 design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Q32 Bio Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A two (2) part study to evaluate the safety, tolerability and PK of ADX-914
Trial website
https://clinicaltrials.gov/show/NCT04485481
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
PI
Address 0 0
Country 0 0
Phone 0 0
1800 243 733
Fax 0 0
Email 0 0
recruitment@nucleusnetwork.com.au
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04485481