Safety Issues
Conditions
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.
Interventions
DRUGADX-914
Single dose from 0.1mg/kg to TBD
DRUGPlacebo
Matching single dose placebo
Sponsors
Q32 Bio Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of subjects with 12 Lead ECG changes | Screening to end of study, up to 18 weeks | Change in 12-lead ECG parameters from baseline to end of study |
| Number of subjects with Clinical safety lab changes | 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 | 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 | Screening to end of study, up to 18 weeks | Listing and change from baseline to end of study |
| Number of subjects with adverse events | Screening to end of study, up to 18 weeks | Listing and summary of AE incidence |
| Number of subjects with Physical exam findings | Screening to end of study, up to 18 weeks | Listing of clinically significant changes in PE findings |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to reach maximum observed plasma concentration, Tmax | Predose to Day 91 (SAD) and Day 127 (MAD) | Of ADX-914 |
| Area Under the plasma concentration time curve, AUC | Predose to Day 91 (SAD) and Day 127 (MAD) | Of ADX-914 |
| Maximum observed plasma concentration, Cmax | Predose to Day 91 (SAD) and Day 127 (MAD) | Of ADX-914 |
Countries
Australia
Outcome results
None listed