Trial Outcomes & Findings for Phase 1 Study of ACE-083 in Healthy Subjects (NCT NCT02257489)
NCT ID: NCT02257489
Last Updated: 2022-09-23
Results Overview
Safety/tolerability assessment, following intramuscular administration, includes adverse events, injection site reactions, laboratory measurements, vital signs, etc.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
58 participants
Primary outcome timeframe
From initiation of treatment (Study Day 1) to end of follow up period (up to Study Day 106)
Results posted on
2022-09-23
Participant Flow
Participant milestones
| Measure |
50 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
6
|
6
|
6
|
6
|
6
|
10
|
6
|
|
Overall Study
COMPLETED
|
6
|
6
|
6
|
5
|
6
|
6
|
6
|
10
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
50 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
0
|
0
|
Baseline Characteristics
Phase 1 Study of ACE-083 in Healthy Subjects
Baseline characteristics by cohort
| Measure |
200 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
n=10 Participants
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
n=6 Participants
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
Total
n=58 Participants
Total of all reporting groups
|
50 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Age, Continuous
|
55.0 years
STANDARD_DEVIATION 2.37 • n=5 Participants
|
55.2 years
STANDARD_DEVIATION 3.71 • n=4 Participants
|
57.0 years
STANDARD_DEVIATION 2.45 • n=21 Participants
|
55.7 years
STANDARD_DEVIATION 3.08 • n=10 Participants
|
57.0 years
STANDARD_DEVIATION 4.73 • n=115 Participants
|
59.2 years
STANDARD_DEVIATION 5.53 • n=24 Participants
|
56.7 years
STANDARD_DEVIATION 7.50 • n=42 Participants
|
56.6 years
STANDARD_DEVIATION 5.2 • n=42 Participants
|
58.0 years
STANDARD_DEVIATION 4.29 • n=5 Participants
|
54.2 years
STANDARD_DEVIATION 9.99 • n=7 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
6 Participants
n=10 Participants
|
6 Participants
n=115 Participants
|
10 Participants
n=24 Participants
|
6 Participants
n=42 Participants
|
58 Participants
n=42 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
1 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
3 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
5 Participants
n=10 Participants
|
6 Participants
n=115 Participants
|
10 Participants
n=24 Participants
|
6 Participants
n=42 Participants
|
55 Participants
n=42 Participants
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
1 Participants
n=42 Participants
|
2 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
6 Participants
n=10 Participants
|
6 Participants
n=115 Participants
|
10 Participants
n=24 Participants
|
5 Participants
n=42 Participants
|
56 Participants
n=42 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: From initiation of treatment (Study Day 1) to end of follow up period (up to Study Day 106)Safety/tolerability assessment, following intramuscular administration, includes adverse events, injection site reactions, laboratory measurements, vital signs, etc.
Outcome measures
| Measure |
50 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
n=10 Participants
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
n=6 Participants
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
ACE-083 Safety and Tolerability: Number of Subjects With Adverse Events
|
6 Participants
|
6 Participants
|
6 Participants
|
6 Participants
|
6 Participants
|
6 Participants
|
6 Participants
|
10 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: PK samples were collected predose, and at 3 hours and 6 hours postdose.Assessment of systemic absorption and exposure following local injection of ACE-083 into the thigh or lower leg muscle
Outcome measures
| Measure |
50 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
n=5 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
ACE-083 Pharmacokinetics: Maximum Measured Plasma Concentrations
Cmax (max) dose 1
|
0 ng/mL (Cmax)
|
168 ng/mL (Cmax)
|
226 ng/mL (Cmax)
|
112 ng/mL (Cmax)
|
219 ng/mL (Cmax)
|
132 ng/mL (Cmax)
|
136 ng/mL (Cmax)
|
—
|
—
|
|
ACE-083 Pharmacokinetics: Maximum Measured Plasma Concentrations
Cmax (min) dose 1
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
0 ng/mL (Cmax)
|
—
|
—
|
SECONDARY outcome
Timeframe: From initiation of treatment (Study Day 1) to end of follow up period (up to Study Day 106)Pharmacodynamic assessments include measurements of thigh or lower leg volume and composition (by MRI) and muscle strength testing (by hand-held dynamometer and fixed system)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: PK samples were collected predose, and at 3 hours and 6 hours postdose.Assessment of systemic absorption and exposure following local injection of ACE-083 into the thigh or lower leg muscle
Outcome measures
| Measure |
50 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
n=5 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
n=6 Participants
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
n=6 Participants
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
ACE-083 Pharmacokinetics: Time of the Maximum Measured Plasma Concentration
Tmax (min) dose 1
|
0 h (Tmax)
|
96 h (Tmax)
|
96 h (Tmax)
|
96 h (Tmax)
|
600 h (Tmax)
|
24.00 h (Tmax)
|
96.0 h (Tmax)
|
—
|
—
|
|
ACE-083 Pharmacokinetics: Time of the Maximum Measured Plasma Concentration
Tmax (max) dose 1
|
0 h (Tmax)
|
96 h (Tmax)
|
168 h (Tmax)
|
96 h (Tmax)
|
600 h (Tmax)
|
96.0 h (Tmax)
|
96.0 h (Tmax)
|
—
|
—
|
Adverse Events
50 mg Single Dose (RF)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
100 mg Single Dose (RF)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
200 mg Single Dose (RF)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
100 mg Multiple Dose (RF)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
200 mg Multiple Dose (RF)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
100 mg Multiple Dose (TA)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
150 mg Multiple Dose (TA)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Pooled Placebo (RF)
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Pooled Placebo (TA)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
50 mg Single Dose (RF)
n=6 participants at risk
8 subjects in total; 6 subjects to receive ACE-083 (50 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Single Dose (RF)
n=6 participants at risk
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Single Dose (RF)
n=6 participants at risk
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, single injection, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (RF)
n=6 participants at risk
8 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
200 mg Multiple Dose (RF)
n=6 participants at risk
8 subjects in total; 6 subjects to receive ACE-083 (200 mg) and 2 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the rectus femoris (RF)
ACE-083: recombinant fusion protein
|
100 mg Multiple Dose (TA)
n=6 participants at risk
9 subjects in total; 6 subjects to receive ACE-083 (100 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
150 mg Multiple Dose (TA)
n=6 participants at risk
9 subjects in total; 6 subjects to receive ACE-083 (150 mg) and 3 subjects to receive placebo, two injections 3 weeks apart, intramuscularly in the tibialis anterior (TA)
ACE-083: recombinant fusion protein
|
Pooled Placebo (RF)
n=10 participants at risk
Pooled placebo from the rectus femoris (RF) cohorts, n=10 (n=2 from each RF cohort)
|
Pooled Placebo (TA)
n=6 participants at risk
Pooled placebo from the tibialis anterior cohorts, n=6 (n=3 from each TA cohort)
|
|---|---|---|---|---|---|---|---|---|---|
|
General disorders
Injection site pain
|
83.3%
5/6
|
83.3%
5/6
|
100.0%
6/6
|
83.3%
5/6
|
100.0%
6/6
|
83.3%
5/6
|
100.0%
6/6
|
100.0%
10/10
|
100.0%
6/6
|
|
Ear and labyrinth disorders
Ear pain
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Injection site haemorrhage
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Injection site reaction
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
50.0%
3/6
|
0.00%
0/6
|
16.7%
1/6
|
10.0%
1/10
|
0.00%
0/6
|
|
General disorders
Injection site discomfort
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
50.0%
3/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
10.0%
1/10
|
50.0%
3/6
|
|
General disorders
Fatigue
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Chills
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Gait disturbance
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
General disorders
Injection site erythema
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Injection site warmth
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
33.3%
2/6
|
20.0%
2/10
|
16.7%
1/6
|
|
General disorders
Pain
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Injection site hypoaesthesia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
General disorders
Injection site paresthesia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
General disorders
Discomfort
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
33.3%
2/6
|
0.00%
0/10
|
33.3%
2/6
|
|
General disorders
Injection site oedema
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
16.7%
1/6
|
0.00%
0/10
|
16.7%
1/6
|
|
General disorders
Pyrexia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Injection site pruritus
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Local swelling
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
General disorders
Feeling hot
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
16.7%
1/6
|
|
Infections and infestations
Oral herpes
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Injury, poisoning and procedural complications
Nerve injury
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Muscle twitching
|
0.00%
0/6
|
16.7%
1/6
|
33.3%
2/6
|
50.0%
3/6
|
33.3%
2/6
|
0.00%
0/6
|
0.00%
0/6
|
30.0%
3/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
1/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
33.3%
2/6
|
16.7%
1/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
66.7%
4/6
|
33.3%
2/6
|
100.0%
6/6
|
100.0%
6/6
|
20.0%
2/10
|
83.3%
5/6
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
0.00%
0/6
|
33.3%
2/6
|
10.0%
1/10
|
16.7%
1/6
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
16.7%
1/6
|
16.7%
1/6
|
10.0%
1/10
|
66.7%
4/6
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
0.00%
0/6
|
0.00%
0/6
|
50.0%
3/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
20.0%
2/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
33.3%
2/6
|
33.3%
2/6
|
10.0%
1/10
|
33.3%
2/6
|
|
Musculoskeletal and connective tissue disorders
Muscle fatigue
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Nervous system disorders
Headache
|
50.0%
3/6
|
16.7%
1/6
|
33.3%
2/6
|
50.0%
3/6
|
0.00%
0/6
|
66.7%
4/6
|
33.3%
2/6
|
10.0%
1/10
|
16.7%
1/6
|
|
Nervous system disorders
Paresthesia
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Nervous system disorders
Migraine
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Nervous system disorders
Sinus headache
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Nervous system disorders
Sensory disturbance
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Nervous system disorders
Aphonia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Nervous system disorders
Lethargy
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Nervous system disorders
Hypoaesthesia
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
33.3%
2/6
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
0.00%
0/6
|
50.0%
3/6
|
16.7%
1/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/6
|
0.00%
0/6
|
50.0%
3/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
16.7%
1/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
50.0%
3/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
33.3%
2/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Dry throat
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Reproductive system and breast disorders
Breast tenderness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
33.3%
2/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Generalised erythema
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Skin odour abnormal
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
10.0%
1/10
|
0.00%
0/6
|
|
Skin and subcutaneous tissue disorders
Skin tightness
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Ear and labyrinth disorders
Ear discomfort
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Eye disorders
Dry eye
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Eye disorders
Eye irritation
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
16.7%
1/6
|
0.00%
0/6
|
0.00%
0/10
|
0.00%
0/6
|
|
Gastrointestinal disorders
Gingival pain
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
16.7%
1/6
|
|
Metabolism and nutrition disorders
Increased appetite
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/6
|
0.00%
0/10
|
16.7%
1/6
|
Additional Information
Senior Vice President, Global Clinical Development
Merck Sharp & Dohme LLC
Phone: 1-800-672-6372
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER