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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04485481




Registration number
NCT04485481
Ethics application status
Date submitted
21/07/2020
Date registered
24/07/2020
Date last updated
28/07/2022

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
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
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
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
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
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
Timepoint [6] 0 0
Screening to end of study, up to 18 weeks
Secondary outcome [1] 0 0
Maximum observed plasma concentration, Cmax
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
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
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
Sex
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/01/2022
Sample size
Target
Accrual to date
Final
42
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/ct2/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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04485481