Trial Outcomes & Findings for Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL) (NCT NCT03514420)
NCT ID: NCT03514420
Last Updated: 2021-02-16
Results Overview
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
COMPLETED
PHASE2
4 participants
Baseline and End of the Treatment (Week 27)
2021-02-16
Participant Flow
This study was conducted at a single center in the United States of America from 15 June 2018 to 21 August 2019.
A total of 8 participants diagnosed with familial partial lipodystrophy (FPL) were screened, out of which 4 participants were treated with AKCEA-ANGPTL3-LRx 20 milligrams (mg). All 4 participants completed the study.
Participant milestones
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by subcutaneous (SC) injection.
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Number analyzed signifies the number of participants with data available for fasting VLDL-C.
Baseline characteristics by cohort
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
42.3 years
STANDARD_DEVIATION 3.77 • n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=4 Participants
|
|
Fasting Triglycerides (TG)
|
817.8 milligram per deciliter (mg/dL)
STANDARD_DEVIATION 431.89 • n=4 Participants
|
|
Fasting Apolipoprotein B-48 (Apo B-48)
|
5.109 mg/dL
STANDARD_DEVIATION 0.5588 • n=4 Participants
|
|
Fasting Apolipoprotein C-III (Apo CIII)
|
28.435 mg/dL
STANDARD_DEVIATION 11.8481 • n=4 Participants
|
|
Fasting Apolipoprotein B (ApoB)
|
124.95 mg/dL
STANDARD_DEVIATION 24.972 • n=4 Participants
|
|
Fasting Non-High Density Lipoprotein- Cholesterol (Non-HDL-C)
|
223.4 mg/dL
STANDARD_DEVIATION 22.03 • n=4 Participants
|
|
Fasting High Density Lipoprotein- Cholesterol (HDL-C)
|
25.13 mg/dL
STANDARD_DEVIATION 3.473 • n=4 Participants
|
|
Fasting Apolipoprotein A-I (Apo A-1)
|
121.3 mg/dL
STANDARD_DEVIATION 9.67 • n=4 Participants
|
|
Fasting Very low Density Lipoprotein-Cholesterol (VLDL-C)
|
130.3 mg/dL
STANDARD_DEVIATION 22.90 • n=3 Participants • Number analyzed signifies the number of participants with data available for fasting VLDL-C.
|
|
Fasting Low Density Lipoprotein Cholesterol (LDL-C)
|
93.3 mg/dL
STANDARD_DEVIATION 24.38 • n=3 Participants • Number analyzed signifies the number of participants with data available for fasting LDL-C.
|
|
Area Under Curve (AUC) of Plasma Glucose
|
257 milligram/deciliter*minute (mg/dL*min)
STANDARD_DEVIATION 45.2622 • n=4 Participants
|
|
AUC of Serum Insulin
|
42.625 milli international units/ liter*minute
STANDARD_DEVIATION 4.852748 • n=4 Participants
|
|
AUC of Serum C-peptide
|
3.266 nanogram/milliliter*minute (ng/mL*min)
STANDARD_DEVIATION 1.234 • n=3 Participants • Number analyzed signifies the number of participants with data available for AUC of Serum C-peptide.
|
|
AUC of Free Fatty Acid
|
1.57 milliequivalents/liter*min (mEq/L*min)
STANDARD_DEVIATION 0.458 • n=3 Participants • Number analyzed signifies the number of participants with data available for AUC of Free fatty acid.
|
|
AUC of Gastric Inhibitory Polypeptide (GIP)
|
46.02 picograms/milliliter*minute (pg/mL*min)
STANDARD_DEVIATION 38.555 • n=3 Participants • Number analyzed signifies the number of participants with data available for AUC of GIP.
|
|
AUC of Glucagon-like Peptide 1 (GLP-1)
|
23.35 picomoles/liter*minute (pmol/L*min)
STANDARD_DEVIATION 17.065 • n=3 Participants • Number analyzed signifies the number of participants with data available for AUC of GLP-1.
|
|
AUC of Peptide Tyrosine Tyrosine (PYY)
|
80.412 pg/mL*min
STANDARD_DEVIATION 99.559 • n=4 Participants
|
|
AUC of Serum Ghrelin
|
27.955 pg/mL*min
STANDARD_DEVIATION 15.127 • n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
|
-59.9 percent change
Standard Deviation 26.29
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the area under the curve (AUC) of Plasma Glucose was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment
|
-9815.0 mg/dL*min
Standard Deviation 4071.43
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of Serum Insulin was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment
|
3262.0 milli international units per liter*min
Standard Deviation 3775.09
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of Serum C-peptide was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment
|
-192.3 ng/mL*min
Standard Deviation 257.10
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of FFA was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment
|
-60.0 mEq/L*min
Standard Deviation 42.52
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of Serum Ghrelin was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment
|
8479.5 pg/mL*min
Standard Deviation 7241.20
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of Incretin Hormone: GIP was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=2 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment
|
-1694.7 pg/mL*min
Standard Deviation 11752.50
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of Incretin Hormone: GLP-1 was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment
|
1677.3 pmol/L*min
Standard Deviation 11301.79
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
Change from Baseline to Week 27 in the AUC of PYY was assessed.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=3 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment
|
6549.0 pg/mL*min
Standard Deviation 10632.50
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in HDL-C at End of the Treatment
|
2.1 mg/dL
Standard Deviation 6.88
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. LDL-C calculated using ultracentrifugation method.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=1 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in LDL-C at End of the Treatment
|
24.0 mg/dL
Standard Deviation NA
Due to a single analyzed participant, standard deviation was not calculated.
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Total Cholesterol (TC) at End of the Treatment
|
-42.3 mg/dL
Standard Deviation 32.29
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. VLDL-C was calculated using direct test method.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=1 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in VLDL-C at End of the Treatment
|
-32.0 mg/dL
Standard Deviation NA
Due to a single analyzed participant, standard deviation was not calculated.
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Non-HDL-C at End of the Treatment
|
-44.4 mg/dL
Standard Deviation 34.27
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoB at End of the Treatment
|
1.25 mg/dL
Standard Deviation 25.265
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoB-48 at End of the Treatment
|
-3.601 mg/dL
Standard Deviation 1.3284
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment
|
4.433 mg/dL
Standard Deviation 24.6001
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoA-1 at End of the Treatment
|
-20.8 mg/dL
Standard Deviation 22.92
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoC-III at End of the Treatment
|
-13.555 mg/dL
Standard Deviation 9.1220
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Data is not available as the collected Baseline samples were not sufficient to assess this outcome measure.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Data is not available as the collected Baseline samples were not sufficient to assess this outcome measure.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, Overall Number of participants analyzed signifies participants who were evaluable for this outcome measure.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=1 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoC-III: LDL at End of the Treatment
|
-2.110 mg/dL
Standard Deviation NA
Due to a single analyzed participant, standard deviation was not calculated.
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in ApoC-III: HDL at End of the Treatment
|
-9.904 mg/dL
Standard Deviation 5.9174
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment
|
8.4 nanomoles per liter (nmol/L)
Standard Deviation 14.99
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment
|
-0.5779 millimoles per liter (mmol/L)
Standard Deviation 0.8778
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Glycerol Levels at End of the Treatment
|
-8.0 micromoles per liter (μmol/L)
Standard Deviation 3.32
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Day 1 pre-dose fasting assessment. Lipoprotein Particle size included: HDL size, LDL size and VLDL size.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
HDL Size
|
0.1 nanometer (nm)
Standard Deviation 0.44
|
|
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
LDL Size
|
0.2 nanometer (nm)
Standard Deviation 0.48
|
|
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
VLDL Size
|
-8.7 nanometer (nm)
Standard Deviation 7.63
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the last non-missing assessment prior to the first dose of study drug.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment
|
-0.23 percentage of HbA1c
Standard Deviation 0.991
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Due to the low number of participants, data for this outcome measure was not collected.
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Day 1 pre-dose fasting assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Adiponectin at End of the Treatment
|
0.08 microgram per milliliter (µg/mL)
Standard Deviation 0.742
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Day 1 pre-dose fasting assessment
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in and Leptin at End of the Treatment
|
-0.19 nanogram per milliliter (ng/mL)
Standard Deviation 2.650
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the last non-missing assessment prior to the first dose of study drug.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment
|
-1.1400 percentage of hepatic fat
Standard Deviation 6.1107
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the last assessment prior to the first dose of study drug. Change in body fat distribution was measured as right anterior thigh skinfold thickness and right tricep skinfold thickness by Skinfold Thickness.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment
Right Anterior Thigh Skinfold Thickness
|
1.6 millimeter (mm)
Standard Deviation 2.38
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment
Right Tricep Skinfold Thickness
|
-2.1 millimeter (mm)
Standard Deviation 3.76
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Screening assessment. Change in body fat distribution (arm bone mass, arm fat mass, arm lean mass, arm total mass, leg bone mass, leg fat mass, leg lean mass, leg total mass, total bone mass, total fat mass, total lean mass, total total mass , trunk bone mass, trunk fat mass, trunk lean mass and trunk total mass) was measures obtained from DEXA.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Trunk Total Mass
|
-500.0 gram (g)
Standard Deviation 836.66
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Arm Bone Mass
|
11.3 gram (g)
Standard Deviation 19.86
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Arm Fat Mass
|
603.8 gram (g)
Standard Deviation 803.36
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Arm Lean Mass
|
273.3 gram (g)
Standard Deviation 612.04
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Arm Total Mass
|
875.0 gram (g)
Standard Deviation 1384.14
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Leg Bone Mass
|
16.5 gram (g)
Standard Deviation 40.53
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Leg Fat Mass
|
436.8 gram (g)
Standard Deviation 662.80
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Leg Lean Mass
|
-345.8 gram (g)
Standard Deviation 1384.52
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Leg Total Mass
|
125.0 gram (g)
Standard Deviation 1736.62
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Total Bone Mass
|
37.3 gram (g)
Standard Deviation 77.03
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Total Fat Mass
|
1197.3 gram (g)
Standard Deviation 1667.27
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Total Lean Mass
|
-891.5 gram (g)
Standard Deviation 1852.25
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Total Total Mass
|
350.0 gram (g)
Standard Deviation 3183.81
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Trunk Bone Mass
|
23.3 gram (g)
Standard Deviation 35.37
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Trunk Fat Mass
|
183.5 gram (g)
Standard Deviation 296.49
|
|
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
Trunk Lean Mass
|
-728.8 gram (g)
Standard Deviation 590.17
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as Screening assessment. Change in body fat distribution for total Bone mineral density was measures obtained from DEXA.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Changes From Baseline in Body Fat Distribution for Total Bone Mineral Density in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment
|
0.0 gram per centimeter square (g/cm^2)
Standard Deviation 0.07
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Due to the low number of participants, data for this outcome measure was not collected.
The baseline was defined as the last assessment prior to the first dose of study drug.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Due to the low number of participants, data for this outcome measure was not collected.
The baseline was defined as the last assessment prior to the first dose of study drug.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Day 1 pre-dose assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Body Weight at End of the Treatment
|
-1.05 kg
Standard Deviation 3.309
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the Screening assessment.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Waist Circumference at End of the Treatment
|
-1.88 cm
Standard Deviation 1.863
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Due to the low number of participants, data for this outcome measure was not collected.
The baseline was defined as screening assessment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as the screening assessment. Quality of life measures the severity of fatigue, severity of trouble thinking or remembering and severity of waking up tired in participants, on a scale ranging from 0 to 3, where, 0= No problem, 1= Mild, 2= Moderate and 3= severe. Higher scores indicates more severity or more impact on quality of life.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Quality of Life (QoL)
Severity of fatigue · Improved
|
1 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of fatigue · Worse
|
1 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of fatigue · No change
|
2 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of trouble thinking or remembering · Improved
|
0 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of trouble thinking or remembering · Worse
|
0 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of trouble thinking or remembering · No change
|
4 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of waking up tired · Improved
|
1 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of waking up tired · Worse
|
2 Participants
|
|
Change From Baseline in Quality of Life (QoL)
Severity of waking up tired · No change
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline and End of the Treatment (Week 27)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
The baseline was defined as a Screening assessment. Pain score is used to determine disease activity in participants, on a scale ranging from 0 to 5 where 0= never, 1= hardly noticed, 2= slightly, 3= moderately, 4= strongly, and 5= very strongly where higher scores indicated higher degree of pain.
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Change From Baseline in Pain Score at End of the Treatment
|
-3.8 score on a scale
Standard Deviation 3.50
|
SECONDARY outcome
Timeframe: From signing of informed consent to end of follow up period (Up to week 40)Population: Safety set included all participants who were enrolled and received at least 1 dose of study drug.
An adverse event (AE) is any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. "A treatment-emergent adverse event (TEAE) is defined as any AE starting on or after the first dose of the study drug
Outcome measures
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 Participants
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
4 Participants
|
Adverse Events
AKCEA-ANGPTL3-LRx 20 mg
Serious adverse events
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 participants at risk
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by subcutaneous (SC) injection.
|
|---|---|
|
Cardiac disorders
Acute coronary syndrome
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Cardiac disorders
Angina pectoris
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Cardiac disorders
Atrial fibrillation
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Cardiac disorders
Atrial flutter
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Cardiac disorders
Ventricular tachycardia
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Troponin increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
Other adverse events
| Measure |
AKCEA-ANGPTL3-LRx 20 mg
n=4 participants at risk
Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by subcutaneous (SC) injection.
|
|---|---|
|
Vascular disorders
Hot flush
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Albumin urine present
|
75.0%
3/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Antinuclear antibody positive
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Gamma-glutamyltransferase increased
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Hepatobiliary disorders
Hepatic steatosis
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood magnesium decreased
|
75.0%
3/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Creatinine urine increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood bilirubin increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
General disorders
Fatigue
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
International normalised ratio increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood glucose increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Glycosylated haemoglobin increased
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Infections and infestations
Conjunctivitis
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
General disorders
Oedema peripheral
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Insulin C-peptide decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Urine albumin/creatinine ratio increased
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Psychiatric disorders
Depression
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Infections and infestations
Gastroenteritis viral
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
urinary casts
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood creatinine increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Nervous system disorders
Presyncope
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Mean cell haemoglobin decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Haemoglobin decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Mean cell volume decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Glomerular filtration rate decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
General disorders
Injection site dryness
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Immune system disorders
Hypersensitivity
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Infections and infestations
Nasopharyngitis
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Alanine aminotransferase increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood alkaline phosphatase increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood calcium decreased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Blood pressure increased
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Crystal urine present
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Investigations
Liver function test
|
50.0%
2/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4 • From signing of informed consent to end of follow up period (Up to week 40)
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo
- Publication restrictions are in place
Restriction type: OTHER