Trial Outcomes & Findings for AXER-204 in Participants With Chronic Spinal Cord Injury (NCT NCT03989440)
NCT ID: NCT03989440
Last Updated: 2023-08-22
Results Overview
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
52 participants
Primary outcome timeframe
Up to Day 29 for Part 1 and Day 253 for Part 2
Results posted on
2023-08-22
Participant Flow
Part 1: 25 participants were enrolled. One patient was withdrawn from the study prior to dosing and is not included in the Safety Population used for data analyses. Part 2: 27 participants were enrolled.
Participant milestones
| Measure |
Part 1 - AXER-204 - 3 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
6
|
7
|
14
|
13
|
|
Overall Study
COMPLETED
|
6
|
6
|
5
|
6
|
14
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
1
|
0
|
0
|
Reasons for withdrawal
| Measure |
Part 1 - AXER-204 - 3 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Overall Study
COVID-19 travel restrictions
|
0
|
0
|
1
|
0
|
0
|
0
|
|
Overall Study
Unsuccessful lumbar puncture
|
0
|
0
|
0
|
1
|
0
|
0
|
Baseline Characteristics
AXER-204 in Participants With Chronic Spinal Cord Injury
Baseline characteristics by cohort
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
n=13 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
Total
n=51 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
00 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
12 Participants
n=8 Participants
|
50 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
1 Participants
n=8 Participants
|
|
Age, Continuous
|
31.7 years
STANDARD_DEVIATION 9.1 • n=5 Participants
|
44.8 years
STANDARD_DEVIATION 11.5 • n=7 Participants
|
37.3 years
STANDARD_DEVIATION 16 • n=5 Participants
|
41.2 years
STANDARD_DEVIATION 16.2 • n=4 Participants
|
41.1 years
STANDARD_DEVIATION 13.8 • n=21 Participants
|
34.5 years
STANDARD_DEVIATION 13.2 • n=8 Participants
|
38.3 years
STANDARD_DEVIATION 13.5 • n=8 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
3 Participants
n=8 Participants
|
10 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
13 Participants
n=21 Participants
|
10 Participants
n=8 Participants
|
41 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
3 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
2 Participants
n=8 Participants
|
7 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
11 Participants
n=8 Participants
|
41 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
6 participants
n=5 Participants
|
6 participants
n=4 Participants
|
14 participants
n=21 Participants
|
13 participants
n=8 Participants
|
51 participants
n=8 Participants
|
|
International Standards for the Neurological Classification of Spinal Cord Injury, Bilateral Upper E
|
24.2 units on a scale
STANDARD_DEVIATION 6.6 • n=5 Participants
|
26.7 units on a scale
STANDARD_DEVIATION 6.3 • n=7 Participants
|
28.0 units on a scale
STANDARD_DEVIATION 5.5 • n=5 Participants
|
28.7 units on a scale
STANDARD_DEVIATION 4.8 • n=4 Participants
|
26.6 units on a scale
STANDARD_DEVIATION 5.4 • n=21 Participants
|
23.9 units on a scale
STANDARD_DEVIATION 5.6 • n=8 Participants
|
26.1 units on a scale
STANDARD_DEVIATION 5.6 • n=8 Participants
|
|
Graded Redefined Assessment of Strength, Sensibility, and Prehension; Bilateral Prehension Performan
|
16.5 units on a scale
STANDARD_DEVIATION 5.8 • n=5 Participants
|
21.0 units on a scale
STANDARD_DEVIATION 7.6 • n=7 Participants
|
18.0 units on a scale
STANDARD_DEVIATION 6.3 • n=5 Participants
|
16.3 units on a scale
STANDARD_DEVIATION 6.7 • n=4 Participants
|
16.4 units on a scale
STANDARD_DEVIATION 3.8 • n=21 Participants
|
18.5 units on a scale
STANDARD_DEVIATION 4.5 • n=8 Participants
|
17.7 units on a scale
STANDARD_DEVIATION 5.4 • n=8 Participants
|
|
Spinal Cord Independence Measure, Version III; Self-care
|
9.8 units on a scale
STANDARD_DEVIATION 5.0 • n=5 Participants
|
8.5 units on a scale
STANDARD_DEVIATION 4.7 • n=7 Participants
|
11.5 units on a scale
STANDARD_DEVIATION 2.8 • n=5 Participants
|
10.3 units on a scale
STANDARD_DEVIATION 3.6 • n=4 Participants
|
10.4 units on a scale
STANDARD_DEVIATION 5.7 • n=21 Participants
|
11 units on a scale
STANDARD_DEVIATION 3.6 • n=8 Participants
|
10.4 units on a scale
STANDARD_DEVIATION 4.4 • n=8 Participants
|
PRIMARY outcome
Timeframe: Up to Day 29 for Part 1 and Day 253 for Part 2An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
n=13 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
|
5 Participants
|
4 Participants
|
5 Participants
|
6 Participants
|
14 Participants
|
10 Participants
|
PRIMARY outcome
Timeframe: Part 1: pre-dose and 1 h, 6 h, 12 h, and 24 h post-dose, Day 4, 8, 15, and 29, Part 2: pre-dose and 4 h post-dose on days 1, 21, 42, 63, and 104; and on Study Days 169 and 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of AXER-204 in Serum
|
NA h*ng/mL
Standard Deviation NA
All values below limit of quantification
|
NA h*ng/mL
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
3670 h*ng/mL
Standard Deviation 1520
|
20900 h*ng/mL
Standard Deviation 7540
|
NA h*ng/mL
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose and 1 h, 6 h, 12 h, and 24 h post-dose, Day 4, 8, 15, and 29, Part 2: pre-dose and 4 h post-dose on days 1, 21, 42, 63, and 104; and on Study Days 169 and 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14)
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Cmax in Serum
|
NA ng/mL
Standard Deviation NA
All values below limit of quantification
|
NA ng/mL
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit determination
|
277 ng/mL
Standard Deviation 79.1
|
641 ng/mL
Standard Deviation 173
|
NA ng/mL
Standard Deviation NA
Could not be determined due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose and 1 h, 6 h, 12 h, and 24 h post-dose, Day 4, 8, 15, and 29, Part 2: pre-dose and 4 h post-dose on days 1, 21, 42, 63, and 104; and on Study Days 169 and 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Tmax in Serum
|
NA hours
Standard Deviation NA
All values below limit of quantification
|
NA hours
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit determination
|
16.8 hours
Standard Deviation 6.5
|
12.8 hours
Standard Deviation 5.06
|
NA hours
Standard Deviation NA
Could not be determined due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose and 1 h, 6 h, 12 h, and 24 h post-dose, Day 4, 8, 15, and 29, Part 2: pre-dose and 4 h post-dose on days 1, 21, 42, 63, and 104; and on Study Days 169 and 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
t1/2 in Serum
|
NA hours
Standard Deviation NA
All values below limit of quantification
|
NA hours
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
NA hours
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
53.9 hours
Standard Deviation 24.8
|
NA hours
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Day 1 pre-dose up to Day 29 in Part 1, Pre-dose up to Day 253 in Part 2Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Clearance From Serum
|
NA L/h
Standard Deviation NA
All values below limit of quantification
|
NA L/h
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
NA L/h
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
4.84 L/h
Standard Deviation 1.41
|
NA L/h
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose and 1 h, 6 h, 12 h, and 24 h post-dose, Day 4, 8, 15, and 29, Part 2: pre-dose and 4 h post-dose on days 1, 21, 42, 63, and 104; and on Study Days 169 and 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Volume of distribution calculated from serum exposure data
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Volume of Distribution
|
NA L
Standard Deviation NA
All values below limit of quantification
|
NA L
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
NA L
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
344 L
Standard Deviation 70.1
|
NA L
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose, post-dose at 24 h, 72 h, Days 8 and 29, Part 2: pre-dose on Days 1, 21, 42, 63, and 104, and on Day 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of AXER-204 in CSF
|
57200 h*ng/mL
Standard Deviation 61500
|
5810000 h*ng/mL
Standard Deviation 12300000
|
12500000 h*ng/mL
Standard Deviation 18000000
|
10000000 h*ng/mL
Standard Deviation 6260000
|
NA h*ng/mL
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose, post-dose at 24 h, 72 h, Days 8 and 29, Part 2: pre-dose on Days 1, 21, 42, 63, and 104, and on Day 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Cmax of AXER-204 in CSF
|
3340 ng/mL
Standard Deviation 2890
|
87900 ng/mL
Standard Deviation 128000
|
280000 ng/mL
Standard Deviation 221000
|
412000 ng/mL
Standard Deviation 129000
|
NA ng/mL
Standard Deviation NA
Could not be determined due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose, post-dose at 24 h, 72 h, Days 8 and 29, Part 2: pre-dose on Days 1, 21, 42, 63, and 104, and on Day 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Tmax of AXER-204 in CSF
|
24.2 h
Standard Deviation 0.91
|
23.9 h
Standard Deviation 0.89
|
23.2 h
Standard Deviation 0.49
|
22.9 h
Standard Deviation 0.93
|
NA h
Standard Deviation NA
Could not be determined due to the limited sampling interval
|
—
|
PRIMARY outcome
Timeframe: Part 1: pre-dose, post-dose at 24 h, 72 h, Days 8 and 29, Part 2: pre-dose on Days 1, 21, 42, 63, and 104, and on Day 253.Population: In Part 2, only pharmacokinetic samples from participants receiving AXER-204 were analyzed (n=14).
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
t1/2 of AXER-204 in CSF
|
NA h
Standard Deviation NA
Quantifiable concentrations were present in too few of the samples to permit calculation
|
23.1 h
Standard Deviation 32.2
|
13.5 h
Standard Deviation 10.8
|
12.5 h
Standard Deviation 7.08
|
NA h
Standard Deviation NA
Could not be calculated due to the limited sampling interval
|
—
|
SECONDARY outcome
Timeframe: Baseline to Day 169Population: This was pre-specified to be an exploratory outcome in Part 1, and therefore data will not be reported
The ISNCSCI bilateral Upper Extremity Motor Score (UEMS) is determined by examining the muscle function within each of the 5 myotomes encompassing arm and hand function on each side of the body. A score ranging from 0 to 5 can be given to each myotome tested resulting in a maximum score of 50. Higher values indicate greater strength.
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=14 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=13 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Change in International Standards for Neurological Classification of SCI (ISNCSCI) Bilateral Upper Extremity Motor Score (UEMS)
|
2.05 score on a scale
Interval 0.59 to 3.52
|
1.52 score on a scale
Interval 0.021 to 3.01
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Day 169Population: This was pre-specified to be an exploratory outcome in Part 1, and therefore data will not be reported
The GRASSP Bilateral Prehension Performance Score is determined based on performance of four tasks with each hand with scores ranging from 0 to 5 for each task resulting in a maximum score of 40. Higher scores indicate better function.
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=14 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=13 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Change in Graded Redefined Assessment of Strength, Sensation and Prehension (GRASSP) Bilateral Prehension Performance Score
|
0.42 score on a scale
Interval -1.07 to 1.91
|
1.97 score on a scale
Interval 0.41 to 3.54
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Day 169Population: This was pre-specified to be an exploratory outcome in Part 1, and therefore data will not be reported
The SCIM III questionnaire self-care score assesses activities of daily living including feeding, bathing, dressing, and grooming. The self-care score ranges 0-20 with higher scores correspond to better ability to carry out these self-care activities.
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=14 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=13 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Change in Version III of the Spinal Cord Independence Measure (SCIM III) Self-care
|
-0.25 score on a scale
Interval -1.5 to 1.0
|
0.91 score on a scale
Interval -0.38 to 2.2
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 169The PGIC instrument captures the patient's overall evaluation of response to treatment. Specifically, the PGIC asks: "Since beginning this clinical trial, how would you describe the overall change (if any) related to your chronic spinal cord injury?" The patient is asked to report the degree to which they have changed since entering the treatment period using a 7-point Likert scale (1='Much worse', 2='Worse', 3='A little worse', 4='No change', 5='A little better', 6='Better', 7='Much better') and "If better or worse, what has changed?". Patients that have evaluation results including "Much better", "Better", or "A little better" are considered Responders.
Outcome measures
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=5 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 Participants
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
n=13 Participants
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Patient Global Impression of Change (PGIC) Responder Rate
|
NA Participants
Not evaluated in Part 1
|
NA Participants
Not evaluated in Part 1
|
NA Participants
Not evaluated in Part 1
|
NA Participants
Not evaluated in Part 1
|
3 Participants
|
4 Participants
|
Adverse Events
Part 1 - AXER-204 - 3 mg
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Part 1 - AXER-204 - 30 mg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Part 1 - AXER-204 - 90 mg
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Part 1 - AXER-204 - 200 mg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Part 2 - AXER-204 - 200 mg
Serious events: 4 serious events
Other events: 14 other events
Deaths: 0 deaths
Part 2 - Placebo
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 participants at risk
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
n=13 participants at risk
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
15.4%
2/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Infections and infestations
Cellulitis
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Renal and urinary disorders
Ureterolithiasis
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
Other adverse events
| Measure |
Part 1 - AXER-204 - 3 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 30 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 90 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 1 - AXER-204 - 200 mg
n=6 participants at risk
Part 1 Open-label single ascending dose. Participants received a single dose of AXER-204. AXER-204: human NoGo Trap fusion protein
|
Part 2 - AXER-204 - 200 mg
n=14 participants at risk
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. AXER-204: human NoGo Trap fusion protein
|
Part 2 - Placebo
n=13 participants at risk
Part 2 Double-blind, placebo-controlled repeat dose. Participants received up to 6 doses given approximately every 21 days. Placebo: Phosphate buffered saline formulation
|
|---|---|---|---|---|---|---|
|
Nervous system disorders
Headache
|
50.0%
3/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
50.0%
3/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
83.3%
5/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
92.9%
13/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
46.2%
6/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
33.3%
2/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
33.3%
2/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
21.4%
3/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
38.5%
5/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Pleocytosis
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
42.9%
6/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Muscle spasticity
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
28.6%
4/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Neuralgia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Neuropathy peripheral
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Syncope
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Hypertonia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Nervous system disorders
Neurological symptom
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
42.9%
6/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
15.4%
2/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Infections and infestations
Cellulitis
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Infections and infestations
Bacterial disease carrier
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Infections and infestations
Corona virus infection
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
15.4%
2/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Anal incontinence
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Odynophagia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
21.4%
3/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
30.8%
4/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Autonomic dysreflexia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
23.1%
3/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Cartilage injury
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Scratch
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Stoma site pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Procedural headache
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
Blood pressure increased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
CSF protein increased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
Body temperature decreased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
CSF white blood cell count increased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Investigations
Blood creatine phosphokinase increased
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Blister
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Blood blister
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Ingrown hair
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Acne
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Pyrexia
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
33.3%
2/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
33.3%
2/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Chills
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Fatigue
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Application site irritation
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Axillary pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Vaccination site pain
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Asthenia
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Feeling cold
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Feeling hot
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Injection site discomfort
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Puncture site bruise
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
General disorders
Swelling
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
16.7%
1/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Psychiatric disorders
Mental fatigue
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
14.3%
2/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Cardiac disorders
Atrioventricular block first degree
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.7%
1/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Vascular disorders
Hypertensive urgency
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
|
Vascular disorders
Labile hypertension
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/6 • Part 1: up to Day 29, Part 2: up to Day 253
|
7.1%
1/14 • Part 1: up to Day 29, Part 2: up to Day 253
|
0.00%
0/13 • Part 1: up to Day 29, Part 2: up to Day 253
|
Additional Information
George D. Maynard, Ph.D., Study Director
ReNetX Bio, Inc.
Phone: 2032088925
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee PIs are free to publish, present, or use institution specific study data and results arising out of their performance of the protocol. At least forty five (45) days prior to submission for publication, PIs must submit to sponsor for review and comment any proposed oral or written publication. The first publication of the results shall be a joint multi-center publication of the study results managed and coordinated by sponsor unless this is not completed within 18 months after completion date.
- Publication restrictions are in place
Restriction type: OTHER