Trial Outcomes & Findings for A Study of JR-171 in Patients With Mucopolysaccharidosis I (NCT NCT04227600)
NCT ID: NCT04227600
Last Updated: 2025-03-19
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
18 participants
Primary outcome timeframe
Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2).
Results posted on
2025-03-19
Participant Flow
Participant milestones
| Measure |
Part1, Dose-escalation
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part 2, Dose-escalation
JR-171, Dose-escalation:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
Participants are included as 4.0 mg/kg/week group.
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
4
|
2
|
6
|
6
|
|
Overall Study
COMPLETED
|
4
|
2
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Baseline characteristics by cohort
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part 2, Dose-escalation
n=2 Participants
JR-171, Dose-escalation:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
Participants are included as 4.0 mg/kg/week group.
|
Part2, 2.0 mg
n=6 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=6 Participants
JR-171, 4.0 mg/kg/week group
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
46.8 years
STANDARD_DEVIATION 13.6 • n=5 Participants
|
NA years
STANDARD_DEVIATION NA • n=7 Participants
|
11.0 years
STANDARD_DEVIATION 6.0 • n=5 Participants
|
12.3 years
STANDARD_DEVIATION 11.4 • n=4 Participants
|
19.2 years
STANDARD_DEVIATION 17.8 • n=21 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Asian (Japanese)
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Asian (Except for Japanese)
|
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
|
|
Race/Ethnicity, Customized
Asian (Total)
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
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
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
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
|
|
Race/Ethnicity, Customized
White
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
2 participants
n=21 Participants
|
|
Region of Enrollment
Japan
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
4 participants
n=21 Participants
|
|
Region of Enrollment
Brazil
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
5 participants
n=5 Participants
|
5 participants
n=4 Participants
|
12 participants
n=21 Participants
|
|
Phenotype
Hurler
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
7 Participants
n=21 Participants
|
|
Phenotype
Hurler-Scheie
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Phenotype
Scheie
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
|
Prior use of laronidase
Yes
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
17 Participants
n=21 Participants
|
|
Prior use of laronidase
No
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
HSCT treatment
Yes
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
HSCT treatment
No
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
17 Participants
n=21 Participants
|
|
Intellectual disabilities
Yes
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Intellectual disabilities
No
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Prior laronidase-related IAR
Yes
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Prior laronidase-related IAR
No
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
15 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2).Population: Since participants with dose-escalation treatment continued treatment with 4.0 mg/kg/week after the first three weeks, they were analyzed as 4.0 mg/kg/week group participants
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=6 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=8 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Number of Participants With Adverse Events
Adverse events
|
3 participants
|
6 participants
|
7 participants
|
|
Number of Participants With Adverse Events
Serious adverse events
|
0 participants
|
1 participants
|
1 participants
|
|
Number of Participants With Adverse Events
Adverse events of special interests
|
1 participants
|
3 participants
|
3 participants
|
|
Number of Participants With Adverse Events
Adverse events leading to death
|
0 participants
|
1 participants
|
0 participants
|
|
Number of Participants With Adverse Events
IARs (Adverse events)
|
1 participants
|
1 participants
|
3 participants
|
|
Number of Participants With Adverse Events
Adverse drug reactions
|
1 participants
|
1 participants
|
3 participants
|
|
Number of Participants With Adverse Events
Serious adverse drug reactions
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events
Adverse drug reactions of special interests
|
1 participants
|
1 participants
|
3 participants
|
|
Number of Participants With Adverse Events
Adverse drug reactions leading to death
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Adverse Events
IARs (Adverse drug reactions)
|
1 participants
|
1 participants
|
3 participants
|
SECONDARY outcome
Timeframe: Week 1 to Week 4Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Pharmacokinetics of Each Dose in Part 1 (AUC0-t)
0.1 mg/kg
|
475.141 h*ng/mL
Standard Deviation 494.371
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (AUC0-t)
1.0 mg/kg
|
10293.474 h*ng/mL
Standard Deviation 8199.941
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (AUC0-t)
2.0 mg/kg
|
30776.649 h*ng/mL
Standard Deviation 28504.162
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (AUC0-t)
4.0 mg/kg
|
107077.423 h*ng/mL
Standard Deviation 89015.623
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 1 to Week 4Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Pharmacokinetics of Each Dose in Part 1 (Cmax)
0.1 mg/kg
|
94.875 ng/mL
Standard Deviation 93.331
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (Cmax)
1.0 mg/kg
|
2825.750 ng/mL
Standard Deviation 2132.838
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (Cmax)
2.0 mg/kg
|
6889.000 ng/mL
Standard Deviation 5195.335
|
—
|
—
|
|
Pharmacokinetics of Each Dose in Part 1 (Cmax)
4.0 mg/kg
|
19040.000 ng/mL
Standard Deviation 13064.037
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 1 to Week 12Population: There is no data for participants in dose-escalation group for plasma drug concentration after administration of JR-171. Though number of participants for PK set is 6 for each group, two participants from the 2.0 mg group and three participants from the 4.0 mg group was excluded from the data set since PK sample was collected by the same route as JR-171 administration and potential contamination could not be ruled out.
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=3 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Pharmacokinetics of Each Dose in Part 2 (AUC0-t)
Week 1
|
18232.756 h*ng/mL
Standard Deviation 9950.396
|
53691.447 h*ng/mL
Standard Deviation 25202.412
|
—
|
|
Pharmacokinetics of Each Dose in Part 2 (AUC0-t)
Week 4
|
19466.227 h*ng/mL
Standard Deviation 7542.104
|
33209.686 h*ng/mL
Standard Deviation 18465.369
|
—
|
|
Pharmacokinetics of Each Dose in Part 2 (AUC0-t)
Week 12
|
22203.369 h*ng/mL
Standard Deviation 7851.121
|
35128.134 h*ng/mL
Standard Deviation 8312.799
|
—
|
SECONDARY outcome
Timeframe: Week 1 to Week 12Population: There is no data for participants in dose-escalation group for plasma drug concentration after administration of JR-171. Though number of participants for PK set is 6 for each group, two participants from the 2.0 mg group and three participants from the 4.0 mg group was excluded from the data set since PK sample was collected by the same route as JR-171 administration and potential contamination could not be ruled out.
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=3 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Pharmacokinetics of Each Dose in Part 2 (Cmax)
Week 1
|
5032.500 ng/mL
Standard Deviation 2755.085
|
11526.667 ng/mL
Standard Deviation 3082.229
|
—
|
|
Pharmacokinetics of Each Dose in Part 2 (Cmax)
Week 4
|
5421.200 ng/mL
Standard Deviation 2094.819
|
8116.667 ng/mL
Standard Deviation 3459.947
|
—
|
|
Pharmacokinetics of Each Dose in Part 2 (Cmax)
Week 12
|
6059.747 ng/mL
Standard Deviation 1889.616
|
8983.333 ng/mL
Standard Deviation 938.740
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 4Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
Baseline
|
1131.8 ng/mL
Standard Deviation 532.9
|
—
|
—
|
|
Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
Week 4
|
467.0 ng/mL
Standard Deviation 500.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 4Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
Baseline
|
264.0 ng/mL
Standard Deviation 72.6
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
Week 4
|
213.0 ng/mL
Standard Deviation 135.4
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Since participants with dose-escalation treatment continued treatment with 4.0mg/kg/week after the first three weeks, they were analyzed as 4.0 mg/kg/group participants. Protocol visit was not performed at Week 12 by one participant in 4.0 mg group.
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=8 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
Baseline
|
3239.8 ng/mL
Standard Deviation 2726.1
|
2054.0 ng/mL
Standard Deviation 1542.7
|
—
|
|
Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
Week 12
|
917.5 ng/mL
Standard Deviation 1216.2
|
407.6 ng/mL
Standard Deviation 372.1
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Since participants with dose-escalation treatment continued treatment with 4.0mg/kg/week after the first three weeks, they were analyzed as 4.0 mg/kg/group participants. Protocol visit was not performed at Week 12 by one participant in 4.0 mg group.
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=8 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
Baseline
|
2241.17 ng/mL
Standard Deviation 2994.88
|
679.38 ng/mL
Standard Deviation 451.16
|
—
|
|
Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
Week 12
|
896.80 ng/mL
Standard Deviation 814.82
|
320.31 ng/mL
Standard Deviation 228.30
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 5Population: Contamination happened with Heparin or Heparin-like compounds in one participant at Week 5.
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Serum in Part 1
Baseline
|
358.5 ng/mL
Standard Deviation 52.7
|
—
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 1
Week 2
|
385.8 ng/mL
Standard Deviation 62.5
|
—
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 1
Week 3
|
339.8 ng/mL
Standard Deviation 59.7
|
—
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 1
Week 4
|
311.3 ng/mL
Standard Deviation 86.3
|
—
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 1
Week 5
|
255.7 ng/mL
Standard Deviation 67.3
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 5Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Serum in Part 1
Baseline
|
965.0 ng/mL
Standard Deviation 224.4
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Serum in Part 1
Week 2
|
929.8 ng/mL
Standard Deviation 204.4
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Serum in Part 1
Week 3
|
1000.5 ng/mL
Standard Deviation 200.1
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Serum in Part 1
Week 4
|
958.0 ng/mL
Standard Deviation 207.1
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Serum in Part 1
Week 5
|
867.3 ng/mL
Standard Deviation 241.3
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separated to S/H and Naive since these two will show a different trend of changes. Contamination happened with Heparin or Heparin-like compounds in 1 participant in 2.0 mg S/H group at W8 HS data were NA for 1 participant at W6(Not assessed), and another participant at W8, 10, 12, and 13(due to hospitalization) in 4.0 mg S/H group. Contamination happened in participant in 4.0 mg Naïve group at BL and W8, and HS data were not available at W4(due to difficult venous access).
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=7 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Serum in Part 2
Baseline
|
668.5 ng/mL
Standard Deviation 341.9
|
465.0 ng/mL
Standard Deviation 314.2
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 2
|
613.3 ng/mL
Standard Deviation 353.9
|
417.9 ng/mL
Standard Deviation 201.0
|
1410 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 4
|
549.8 ng/mL
Standard Deviation 315.9
|
368.9 ng/mL
Standard Deviation 184.8
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 6
|
509.7 ng/mL
Standard Deviation 331.2
|
268.3 ng/mL
Standard Deviation 135.4
|
549 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 8
|
545.2 ng/mL
Standard Deviation 368.2
|
265.5 ng/mL
Standard Deviation 109.6
|
—
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 10
|
538.5 ng/mL
Standard Deviation 357.7
|
281.3 ng/mL
Standard Deviation 131.6
|
522 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 12
|
454.8 ng/mL
Standard Deviation 341.2
|
274.0 ng/mL
Standard Deviation 100.3
|
594 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Serum in Part 2
Week 13
|
487.5 ng/mL
Standard Deviation 306.7
|
312.8 ng/mL
Standard Deviation 202.6
|
491 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separate to S/H and Naive in this table, since these two will show a different trend of changes. DS data were NA for one participant at W6 (Not assessed), and for another participant at Week 8, 10, 12, and 13 (due to hospitalization) in 4.0 mg Switched/HSCT group. DS data were NA at Week 4 due to difficult venous access in participant in 4.0 mg Naïve group.
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=7 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Baseline
|
1664.5 ng/mL
Standard Deviation 670.8
|
1442.4 ng/mL
Standard Deviation 756.6
|
4110 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 2
|
1847.5 ng/mL
Standard Deviation 991.8
|
1571.6 ng/mL
Standard Deviation 916.6
|
1740 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 4
|
1639.3 ng/mL
Standard Deviation 792.1
|
1321.6 ng/mL
Standard Deviation 687.2
|
—
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 6
|
1585.5 ng/mL
Standard Deviation 686.8
|
1234.0 ng/mL
Standard Deviation 807.1
|
788 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 8
|
1553.7 ng/mL
Standard Deviation 811.9
|
1191.5 ng/mL
Standard Deviation 681.7
|
679 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 10
|
1510.2 ng/mL
Standard Deviation 704.1
|
1206.7 ng/mL
Standard Deviation 719.3
|
702 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 12
|
1362.2 ng/mL
Standard Deviation 642.8
|
1208.8 ng/mL
Standard Deviation 763.4
|
899 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Serum in Part 2
Week 13
|
1510.8 ng/mL
Standard Deviation 699.1
|
1197.2 ng/mL
Standard Deviation 811.1
|
762 ng/mL
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
SECONDARY outcome
Timeframe: Baseline to Week 5Population: Contamination happened with Heparin or Heparin-like compounds in one participant at Week 5.
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Urine in Part 1
Baseline
|
31.050 µg/mgCr
Standard Deviation 14.339
|
—
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 1
Week 2
|
34.835 µg/mgCr
Standard Deviation 11.885
|
—
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 1
Week 3
|
27.314 µg/mgCr
Standard Deviation 20.634
|
—
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 1
Week 4
|
21.773 µg/mgCr
Standard Deviation 19.901
|
—
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 1
Week 5
|
20.321 µg/mgCr
Standard Deviation 20.612
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 5Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Urine in Part 1
Baseline
|
22.704 µg/mgCr
Standard Deviation 13.815
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 1
Week 2
|
29.317 µg/mgCr
Standard Deviation 8.615
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 1
Week 3
|
24.366 µg/mgCr
Standard Deviation 16.614
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 1
Week 4
|
20.400 µg/mgCr
Standard Deviation 16.880
|
—
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 1
Week 5
|
17.112 µg/mgCr
Standard Deviation 17.026
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separated into S/H and Naive since it shows a different trend of changes. 4 participants data were excluded from analysis due to incorrect procedure of assessment (2 each for 2.0 and 4.0 mg S/H) Data were NA for 3 participants (1 at W12 (2.0 mg)\[missing creatinine data\] and 1 at W6 \[No assessment\], 1 at W8, 10, 12, 13 (4.0 mg S/H) \[due to hospitalization\]) Contamination happened in participant in 4.0 mg Naïve group at BL, and HS data were NA at W2, 4, 6 (due to creatinine BLD)
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=5 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Heparan Sulfate Concentrations in Urine in Part 2
Baseline
|
120.572 µg/mgCr
Standard Deviation 152.114
|
35.387 µg/mgCr
Standard Deviation 24.919
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 2
|
82.487 µg/mgCr
Standard Deviation 103.439
|
17.962 µg/mgCr
Standard Deviation 10.584
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 4
|
73.752 µg/mgCr
Standard Deviation 99.221
|
10.927 µg/mgCr
Standard Deviation 5.751
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 6
|
89.412 µg/mgCr
Standard Deviation 122.895
|
11.662 µg/mgCr
Standard Deviation 5.623
|
—
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 8
|
90.174 µg/mgCr
Standard Deviation 137.086
|
6.963 µg/mgCr
Standard Deviation 3.027
|
93.24 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 10
|
79.363 µg/mgCr
Standard Deviation 112.278
|
9.211 µg/mgCr
Standard Deviation 2.844
|
120.98 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 12
|
84.446 µg/mgCr
Standard Deviation 128.037
|
10.412 µg/mgCr
Standard Deviation 4.905
|
131.33 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Heparan Sulfate Concentrations in Urine in Part 2
Week 13
|
79.425 µg/mgCr
Standard Deviation 110.272
|
15.642 µg/mgCr
Standard Deviation 5.705
|
124.41 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separated into S/H and Naive since it shows a different trend of changes. 4 participants data were excluded from analysis due to incorrect procedure of assessment (2 each for 2.0 and 4.0 mg S/H) Data were NA for 3 participants (1 at W12 (2.0 mg)\[missing creatinine data\] and 1 at W6 \[No assessment\], 1 at W8, 10, 12, 13 (4.0 mg S/H) \[due to hospitalization\]). DS data were not available at W2, 4, and 6 (due to creatinine BLD)
Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=5 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Baseline
|
130.164 µg/mgCr
Standard Deviation 131.983
|
39.750 µg/mgCr
Standard Deviation 16.704
|
630.43 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 2
|
114.215 µg/mgCr
Standard Deviation 128.204
|
30.817 µg/mgCr
Standard Deviation 12.606
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 4
|
103.212 µg/mgCr
Standard Deviation 117.351
|
20.873 µg/mgCr
Standard Deviation 9.749
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 6
|
117.873 µg/mgCr
Standard Deviation 145.111
|
20.127 µg/mgCr
Standard Deviation 10.079
|
—
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 8
|
117.417 µg/mgCr
Standard Deviation 158.567
|
13.330 µg/mgCr
Standard Deviation 7.224
|
60.61 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 10
|
103.365 µg/mgCr
Standard Deviation 124.166
|
13.171 µg/mgCr
Standard Deviation 3.257
|
78.61 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 12
|
95.576 µg/mgCr
Standard Deviation 131.815
|
15.406 µg/mgCr
Standard Deviation 3.826
|
80.21 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Dermatan Sulfate Concentrations in Urine in Part 2
Week 13
|
97.508 µg/mgCr
Standard Deviation 116.753
|
18.432 µg/mgCr
Standard Deviation 6.375
|
85.04 µg/mgCr
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
SECONDARY outcome
Timeframe: Baseline to Week 5Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Liver Volumes (Body Weight Adjusted) in Part 1
Baseline
|
21.912 cm3/kg
Standard Deviation 3.607
|
—
|
—
|
|
Liver Volumes (Body Weight Adjusted) in Part 1
Week 5
|
22.332 cm3/kg
Standard Deviation 3.916
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 5Outcome measures
| Measure |
Part1, Dose-escalation
n=4 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Spleen Volumes (Body Weight Adjusted) in Part 1
Baseline
|
4.342 cm3/kg
Standard Deviation 2.978
|
—
|
—
|
|
Spleen Volumes (Body Weight Adjusted) in Part 1
Week 5
|
4.914 cm3/kg
Standard Deviation 4.011
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separated to Switched/HSCT and Naive in this table, since these two will show a different trend of changes. Liver volume data were not available for one participant each in 2.0 mg and 4.0 mg Switched/HSCT group (One couldn't arrive at the site because of the weather and another was hospitalized). Another participant data in 4.0 mg Switched/HSCT group was excluded from analysis since assessment was performed out of the acceptable time frame.
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=7 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Liver Volumes (Body Weight Adjusted) in Part 2
Baseline
|
25.027 cm3/kg
Standard Deviation 6.129
|
25.510 cm3/kg
Standard Deviation 12.295
|
35.95 cm3/kg
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Liver Volumes (Body Weight Adjusted) in Part 2
Week 13
|
23.795 cm3/kg
Standard Deviation 6.602
|
20.014 cm3/kg
Standard Deviation 3.778
|
29.08 cm3/kg
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
SECONDARY outcome
Timeframe: Baseline to Week 13Population: Participants are separated to Switched/HSCT and Naive in this table, since these two will show a different trend of changes. Spleen volume data were not available for one participant each in 2.0 mg and 4.0 mg Switched/HSCT group (One couldn't arrive at the site because of the weather and another was hospitalized). Another participant data in 4.0 mg Switched/HSCT group was excluded from analysis since assessment was performed out of the acceptable time frame.
Outcome measures
| Measure |
Part1, Dose-escalation
n=6 Participants
JR-171, Dose-escalation:
* Initial dose, 0.1 mg/kg/week
* Second dose, 1.0 mg/kg/week
* Third dose, 2.0 mg/kg/week
* Fourth dose, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=7 Participants
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=1 Participants
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
|
|---|---|---|---|
|
Spleen Volumes (Body Weight Adjusted) in Part 2
Baseline
|
3.924 cm3/kg
Standard Deviation 0.766
|
4.416 cm3/kg
Standard Deviation 2.084
|
4.30 cm3/kg
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
|
Spleen Volumes (Body Weight Adjusted) in Part 2
Week 13
|
3.629 cm3/kg
Standard Deviation 0.492
|
4.408 cm3/kg
Standard Deviation 1.486
|
3.10 cm3/kg
Standard Deviation NA
Since the group has one participant, the value in Mean is not a mean value but the actual measured value for the participant.
|
Adverse Events
Part1, Dose-escalation
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Part2, 2.0 mg
Serious events: 1 serious events
Other events: 6 other events
Deaths: 1 deaths
Part 2, 4.0 mg
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part1, Dose-escalation
n=4 participants at risk
JR-171, Dose-escalation:
* Initial administration, 0.1 mg/kg/week
* Second administration, 1.0 mg/kg/week
* Third administration, 2.0 mg/kg/week
* Fourth administration, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=6 participants at risk
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=8 participants at risk
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
Since participants with dose-escalation treatment continued treatment with 4.0mg/kg/week after the first three weeks, they were analyzed as 4.0 mg/kg/group participants.
|
|---|---|---|---|
|
Cardiac disorders
Cardiac failure
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
Other adverse events
| Measure |
Part1, Dose-escalation
n=4 participants at risk
JR-171, Dose-escalation:
* Initial administration, 0.1 mg/kg/week
* Second administration, 1.0 mg/kg/week
* Third administration, 2.0 mg/kg/week
* Fourth administration, 4.0 mg/kg/week
|
Part2, 2.0 mg
n=6 participants at risk
JR-171, 2.0 mg/kg/week group
|
Part 2, 4.0 mg
n=8 participants at risk
JR-171, 4.0 mg/kg/week group 4.0 mg/kg/week group includes two participants with dose-escalation treatment for the first three weeks:
* Initial dose, 1.0 mg/kg/week
* Second dose, 2.0 mg/kg/week
* Third dose, 4.0 mg/kg/week
* Subsequent doses, 4.0 mg/kg/week
Since participants with dose-escalation treatment continued treatment with 4.0mg/kg/week after the first three weeks, they were analyzed as 4.0 mg/kg/group participants.
|
|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 2 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Skin and subcutaneous tissue disorders
Stasis dermatitis
|
25.0%
1/4 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 3 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
50.0%
2/4 • Number of events 2 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Musculoskeletal and connective tissue disorders
Spondylolisthesis
|
25.0%
1/4 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
25.0%
1/4 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
General disorders
Pyrexia
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
25.0%
2/8 • Number of events 4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Investigations
Blood iron decreased
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Investigations
CSF pressure increased
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Investigations
International normalised ratio increased
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Investigations
Transaminases increased
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Investigations
Lymphocyte morphology abnormal
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Injury, poisoning and procedural complications
Arthropod sting
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Injury, poisoning and procedural complications
Wound complication
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Influenza
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
33.3%
2/6 • Number of events 2 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Rhinitis
|
25.0%
1/4 • Number of events 2 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
50.0%
3/6 • Number of events 3 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Cutaneous larva migrans
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Infections and infestations
Device related infection
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Blood and lymphatic system disorders
Normocytic anaemia
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Cardiac disorders
Aortic valve incompetence
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Cardiac disorders
Congestive cardiomyopathy
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Vascular disorders
Hypotension
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
25.0%
1/4 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
16.7%
1/6 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
1/4 • Number of events 1 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/4 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
12.5%
1/8 • Number of events 2 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
25.0%
1/4 • Number of events 3 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/6 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
0.00%
0/8 • Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2). Events occurring within 28 days after the time of observation at Week 5 in Part 1/Week 13 in Part 2 or discontinuation that are considered related to JR-171 will also be collected.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place