Trial Outcomes & Findings for Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients (NCT NCT01678898)
NCT ID: NCT01678898
Last Updated: 2023-09-13
Results Overview
Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
18 participants
Primary outcome timeframe
12 months
Results posted on
2023-09-13
Participant Flow
Recruitment efforts were conducted in North and South America, Europe and Australia for PB-102-F01 (3 months), and all patients who completed this study continued to the PB-102-F02 extension study for an additional 9 months, to complete a total of 12 months of treatment. 18 patients were eligible for enrollment, and 16 completed the study.
Eighteen (18) patients were eligible for enrollment for PB-102-F01 study. The eligible patients who completed PB-102-F01, needed to sign an informed consent for PB-102-F02.
Participant milestones
| Measure |
0.2 mg/kg
PRX-102 0.2 mg/kg every 2 weeks
PRX-102
|
1 mg/kg
PRX-102 1 mg/kg every 2 weeks
PRX-102
|
2 mg/kg
PRX-102 2 mg/kg every 2 weeks
PRX-102
|
|---|---|---|---|
|
PB-102-F01
STARTED
|
6
|
8
|
4
|
|
PB-102-F01
COMPLETED
|
6
|
6
|
4
|
|
PB-102-F01
NOT COMPLETED
|
0
|
2
|
0
|
|
PB-102-F02
STARTED
|
6
|
6
|
4
|
|
PB-102-F02
COMPLETED
|
6
|
6
|
4
|
|
PB-102-F02
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
0.2 mg/kg
PRX-102 0.2 mg/kg every 2 weeks
PRX-102
|
1 mg/kg
PRX-102 1 mg/kg every 2 weeks
PRX-102
|
2 mg/kg
PRX-102 2 mg/kg every 2 weeks
PRX-102
|
|---|---|---|---|
|
PB-102-F01
Adverse Event
|
0
|
1
|
0
|
|
PB-102-F01
Physician Decision
|
0
|
1
|
0
|
Baseline Characteristics
A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
Baseline characteristics by cohort
| Measure |
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
PRX-102
|
1 mg/kg
n=8 Participants
PRX-102 1 mg/kg every 2 weeks
PRX-102
|
2 mg/kg
n=4 Participants
PRX-102 2 mg/kg every 2 weeks
PRX-102
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=6 Participants
|
1 Participants
n=8 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=18 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=6 Participants
|
7 Participants
n=8 Participants
|
4 Participants
n=4 Participants
|
17 Participants
n=18 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=6 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=18 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=6 Participants
|
2 Participants
n=8 Participants
|
3 Participants
n=4 Participants
|
7 Participants
n=18 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=6 Participants
|
6 Participants
n=8 Participants
|
1 Participants
n=4 Participants
|
11 Participants
n=18 Participants
|
|
Phenotypically Classic vs Non Classic Fabry Patients
Phenotypically Classic Fabry Patients
|
5 Participants
n=6 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
4 Participants
n=6 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
1 Participants
n=4 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
10 Participants
n=16 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
|
Phenotypically Classic vs Non Classic Fabry Patients
Non Classic Fabry Patients
|
1 Participants
n=6 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
2 Participants
n=6 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
3 Participants
n=4 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
6 Participants
n=16 Participants • A subgroup analysis of patients with phenotypically classic Fabry disease was conducted only in the efficacy group. The efficacy group includes those who completed the study.
|
PRIMARY outcome
Timeframe: 12 monthsReportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment.
Outcome measures
| Measure |
Safety Group
n=18 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=8 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=4 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Adverse Events
|
54 adverse events
|
11 adverse events
|
29 adverse events
|
14 adverse events
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Plasma Gb3 concentration (ug/mL) was measured at baseline and every 3 months up to 12 months.Results are presented as mean percent change from baseline (visit 1) to 12 months +/- standard error.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
n=9 Participants
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Plasma Gb3 Concentrations
|
-16.8 mean percent change
Standard Error 8.6
|
-30.7 mean percent change
Standard Error 11.2
|
-16.2 mean percent change
Standard Error 12.7
|
-22.2 mean percent change
Standard Error 6.1
|
-33.3 mean percent change
Standard Error 7.6
|
OTHER_PRE_SPECIFIED outcome
Timeframe: eGFR is performed at baseline (day 1) weeks 4, 8, 12, 26, 38 and 52 (12 months)Estimated glomerular filtration rate (eGFR) was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Levels of eGFR calculated by the CKD-EPI equation, based on measured serum creatinine on day 1, weeks 4, 8, 12, 26, 38 and 52 are used to determine the annualized slope of eGFR per patient over a 12 months period. The absolute mean change from baseline (visit 1) to 12 months is then derived from the eGFR slope.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Kidney Function - Change in eGFR
|
-0.8 mL/min/1.73 m2
Standard Error 1.9
|
0 mL/min/1.73 m2
Standard Error 2.8
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Plasma Lyso-Gb3 concentration (ng/mL) was measured at baseline and every 3 months up to 12 months.Results are presented as mean percent change from baseline (visit 1) to 12 months +/- standard error.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
n=10 Participants
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Plasma Lyso-Gb3 Levels
|
-43.4 mean percent change
Standard Error 12.2
|
-59.9 mean percent change
Standard Error 7.1
|
-40.4 mean percent change
Standard Error 7.5
|
-48.9 mean percent change
Standard Error 5.7
|
-57.6 mean percent change
Standard Error 6.8
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (day 1) in PB-102-F01 study and after a total of 6 months of treatment (i.e., at 3 months into study PB-102-F02).Population: 3 patients were not included in the renal biopsies Gb3 analysis. 1 male patient's biopsies were mixed up by the lab; 1 female patient's baseline biopsy didn't include cortex tissue making sample unreadable; and 1 male patient carries a cardiac GLA variant (p.N215S) associated with rare renal manifestation and had a low BLISS baseline score
Kidney biopsy was performed at baseline of study PB-102-F01 and following 6 months treatment with PRX-102. Approximately 300 capillaries were scored in each specimen. A quantitative Barisoni Lipid Inclusion Scoring System (BLISS) was used for scoring Gb3 inclusions in kidney peritubular capillary (PTC) biopsy samples.The scoring system was implemented by 3 blinded pathologists/readers.The BLISS scoring methodology consists of counting the number of Gb-3 inclusions per capillary previously annotated.The final score of each biopsy was the average number of inclusions per capillary. A decrease in scoring from baseline to 6 Month is considered an indication for clinical improvement.
Outcome measures
| Measure |
Safety Group
n=13 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=8 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Change in Kidney Gb3 Accumulation
|
-67.8 Percent change
Standard Error 8.9
|
-84.1 Percent change
Standard Error 3.4
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cardiac MRI was performed in order to assess myocardial fibrosis at baseline, 6 months and 12 months visit.Cardiac MRI was performed to estimate the percentage and mass of the myocardial fibrotic area. Results represent the number of subjects with fibrosis after 1 year of treatment.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Cardiac Fibrosis Per MRI
|
0 number of subjects
|
0 number of subjects
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months.Results are presented as mean percent change from baseline (visit 1) to 12 months.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Cardiac MRI - Ejection Fraction
|
-3.1 Percent Change
Standard Error 3.6
|
-7.3 Percent Change
Standard Error 3.2
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months.Results are presented as mean percent change from baseline (visit 1) to 12 months.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Cardiac MRI - LVM
|
0 Percent change
Standard Error 2.5
|
-2.6 Percent change
Standard Error 3.4
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months.Cardiac MRI with computer-calculated left ventricular mass index were conducted at baseline, 6 month, and 12 months. Results are presented as mean percent change from baseline (visit 1) to 12 months. LVMI is calculated by dividing the left ventricular mass by body surface area.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Cardiac MRI - LVMI
|
0.4 Percent Change
Standard Error 2.6
|
-3.1 Percent Change
Standard Error 3.1
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion.PK parameters were derived from the plasma concentration versus time profiles. Results reported represent the averaged values following a single dosing of the study drug.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Pharmacokinetics - AUC
|
70070 ng*hr/ml
Standard Error 26044
|
390896 ng*hr/ml
Standard Error 136476
|
619393 ng*hr/ml
Standard Error 158562
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion.PK parameters were derived from the plasma concentration versus time profiles. Results reported represent the averaged values following a single dosing of the study drug.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Pharmacokinetics - Terminal Half Life
|
60.3 hour
Standard Error 19.6
|
78.9 hour
Standard Error 10.3
|
70.7 hour
Standard Error 18
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion.PK parameters were derived from the plasma concentration versus time profiles. Clearance of drug from plasma represents the volume of plasma cleared of the drug per unit time per Kg. Results reported represent the averaged values following a single dosing of the study drug.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Pharmacokinetics - Clearance of Drug (Cl)
|
2.96 ml/hr/kg
Standard Error 0.81
|
2.85 ml/hr/kg
Standard Error 0.66
|
3.41 ml/hr/kg
Standard Error 0.68
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion.PK parameters were derived from the plasma concentration versus time profiles. Vz is the volume of distribution during the elimination phase. Results reported represent the averaged values following a single dosing of the study drug.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Pharmacokinetics - Volume of Distribution (Vz)
|
246 ml/kg
Standard Error 68
|
321 ml/kg
Standard Error 71
|
345 ml/kg
Standard Error 105
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion.Pharmacokinetic (PK) parameters were derived from the plasma concentration versus time profiles. Cmax is the maximal plasma concentration of a drug after administration. Results reported represent the averaged values following a single dosing of the study drug.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Pharmacokinetics - Cmax
|
1858 ng/ml
Standard Error 531
|
11123 ng/ml
Standard Error 2409
|
16625 ng/ml
Standard Error 4299
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Anti-pegunigalsidase alfa (PRX-102) antibodies, including neutralizing antibodies in patients having a positive IgG antibody response, were assessed at Visit 1, 2 (Month 1), and then every 2 months during the study, and 2 months after the last infusion.Results reported represent the number of participants who were tested positive for anti-pegunigalsidase alfa (PRX-102) antibodies, including neutralizing antibodies in patients having a positive IgG antibody response per group, at Visit 1, 2 (Month 1), and then every 2 months during the study, and 2 months after the last infusion.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Number of Participants With Anti-Drug Antibodies
|
2 participants
|
1 participants
|
0 participants
|
3 participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: The Short Form Brief Pain Inventory (BPI) is assessed at baseline, 3-month, 6-month, and 12-month visits.Pain severity (worst, least, average, and right now) is summarized at baseline, 3-month, 6-month and 12-month of the study. The Short Form Brief Pain Inventory (BPI) is based on a 10-point scale, where 0=no pain and 10=pain as bad as you can imagine. A reduction in pain severity score indicated an improvement. The changes from baseline for individual scores and composite scores (a mean severity score) was assessed. The mean change in score from baseline at the 12-month visit is reflected.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Change in Short Form Brief Pain Inventory (BPI)
|
-0.7 score on a scale
Standard Error 0.4
|
-1.2 score on a scale
Standard Error 0.4
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Urine creatinine level is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visits using spot urine tests.Proteinuria is assessed using spot urine tests at baseline, 3-month, 6-month, 9-month, and 12-month visits. The percent changes of urine creatinine level at the 12-month visit from baseline is reflected.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Urine Creatinine Level
|
-15.9 Percentage change from baseline
Standard Error 25.6
|
-15.3 Percentage change from baseline
Standard Error 27.8
|
-68.3 Percentage change from baseline
Standard Error 2.5
|
-28.8 Percentage change from baseline
Standard Error 14.6
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Protein/.creatinine ratio is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visitsProteinuria is assessed using spot urine tests at baseline, 3-month, 6-month, 9-month, and 12-month visits. The percent changes of urine protein/creatinine ratio at the 12-month visit from baseline is reflected.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Urine Protein/Creatinine Ratio
|
-2.6 Percentage change from baseline
Standard Error 23.4
|
-14.8 Percentage change from baseline
Standard Error 14.1
|
-7.9 Percentage change from baseline
Standard Error 12.4
|
-8.5 Percentage change from baseline
Standard Error 10.1
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Total urine protein level is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visits.Proteinuria is assessed using spot urine tests at baseline, 3-month, 6-month, 9-month, and 12-month visits. The percent changes of total urine protein level at the 12-month visit from baseline is reflected.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Total Urine Protein Level
|
-35.1 Percentage change from baseline
Standard Error 18.4
|
-32.8 Percentage change from baseline
Standard Error 17.4
|
-71.7 Percentage change from baseline
Standard Error 7.3
|
-43.4 Percentage change from baseline
Standard Error 9.9
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Brain MRI is performed at baseline and 12-month visit.Qualitative assessments regarding evidence of stroke using brain MRI were summarized at baseline and 12-month visits. Number is subjects with evidence of stoke on the brain MRI at the 12-month visit is reflected.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Brain MRI
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: The MSSI is performed at baseline, 6-month, and 12-monthThe Mainz Severity Score Index (MSSI) is useful for monitoring clinical improvement in patients receiving enzyme replacement therapy. The MSSI scoring system is composed of four sections that cover the general, neurological,cardiovascular and renal signs and symptoms of Fabry disease. Each section includes a group of signs and symptoms that are associated with Fabry disease. The minimal score is 0, and the maximum score for the general section is 18. A higher score indicates more severe clinical manifestations of the disease.The MSSI is performed at baseline, 6-month, and 12-month. The overall mean reduction of the total general score at 12-month from baseline is reflected.
Outcome measures
| Measure |
Safety Group
n=6 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=6 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
n=4 Participants
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
n=16 Participants
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Change in Mainz Severity Score Index (MSSI)
|
-2.8 score of a scale
Standard Error 1.2
|
-1.2 score of a scale
Standard Error 0.6
|
-1.5 score of a scale
Standard Error 0.9
|
-1.9 score of a scale
Standard Error 0.6
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: The Gastrointestinal Symptoms Questionnaire is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visits.A qualitative assessments regarding abdominal pain and diarrhea at baseline, 3-month, 6-month, 9-month, and 12-month visits. The subjects were asked to report the severity of their abdominal pain as no pain, mild, moderate, severe or really severe. The number of subjects who reported no or mild abdominal pain at the 12-month visit are reflected.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Gastrointestinal Symptoms Questionnaire - Severity of Abdominal Pain
|
10 participants
|
7 participants
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: The Gastrointestinal Symptoms Questionnaire is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visits.A qualitative assessments regarding abdominal pain and diarrhea at baseline, 3-month, 6-month, 9-month, and 12-month visits. The subjects were asked to report their frequency of abdominal pain as never, rarely, monthly, weekly, or daily. The numbers of subjects who reported never or rarely having abdominal pain at the 12-month visit are reflected.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Gastrointestinal Symptoms Questionnaire - Frequency of Abdominal Pain
|
7 Participants
|
5 Participants
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: The Gastrointestinal Symptoms Questionnaire is assessed at baseline, 3-month, 6-month, 9-month, and 12-month visits.A qualitative assessments regarding abdominal pain and diarrhea at baseline, 3-month, 6-month, 9-month, and 12-month visits. The subjects were asked to report their frequency of diarrhea as never, rarely, monthly, weekly, or daily. The number of subjects who reported never or rarely having diarrhea at the 12-month visit is reflected.
Outcome measures
| Measure |
Safety Group
n=16 Participants
The safety population in the study included 18 patients: 6 patients in the 0.2 mg/kg treatment group, 8 patients in the 1.0 mg/kg, and 4 patients in the 2.0 mg/kg treatment groups.
|
0.2 mg/kg
n=10 Participants
PRX-102 0.2 mg/kg every 2 weeks
|
1.0 mg/kg
PRX-102 1.0 mg/kg every 2 weeks
|
2.0 mg/kg
PRX-102 2.0 mg/kg every 2 weeks
|
Phenotypically Classic Fabry Patients
Patients who are phenotypically classic Fabry Patients.
|
|---|---|---|---|---|---|
|
Gastrointestinal Symptoms Questionnaire - Frequency of Diarrhea
|
11 Participants
|
6 Participants
|
—
|
—
|
—
|
Adverse Events
0.2 mg/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
1 mg/kg
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
2 mg/kg
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
0.2 mg/kg
n=6 participants at risk
PRX-102 0.2 mg/kg every 2 weeks
|
1 mg/kg
n=8 participants at risk
PRX-102 1.0 mg/kg every 2 weeks
|
2 mg/kg
n=4 participants at risk
PRX-102 2.0 mg/kg every 2 weeks
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Renal Hematoma
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
Other adverse events
| Measure |
0.2 mg/kg
n=6 participants at risk
PRX-102 0.2 mg/kg every 2 weeks
|
1 mg/kg
n=8 participants at risk
PRX-102 1.0 mg/kg every 2 weeks
|
2 mg/kg
n=4 participants at risk
PRX-102 2.0 mg/kg every 2 weeks
|
|---|---|---|---|
|
Vascular disorders
Hypotension
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 3 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Chest pain
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 3 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
50.0%
2/4 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
2/8 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Chest discomfort
|
16.7%
1/6 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Infusion reaction
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
2/8 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • Number of events 7 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Skin and subcutaneous tissue disorders
Itching
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Skin and subcutaneous tissue disorders
Sweating
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Eye disorders
Corneal Edema
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Eye disorders
Conjunctival Hemorrhage
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Gastrointestinal disorders
Diarrhea
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Edema
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Peripheral Edema
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus hypersecretion
|
16.7%
1/6 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Nervous system disorders
Visual field defect
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Nervous system disorders
Typical aura without headache
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/8 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
1/4 • Number of events 2 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Fatigue
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
25.0%
2/8 • Number of events 6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
General disorders
Pain
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Stiffness
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
|
Psychiatric disorders
Nervousness
|
0.00%
0/6 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
12.5%
1/8 • Number of events 1 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
0.00%
0/4 • AEs were collected from the start of treatment throughout the 12 months of the study, plus a follow-up visit 2 months after 12-months study visit.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place