This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
AKCEA-ANGPTL3-LRx solution for SC injection.
Clinical Site
Ann Arbor, Michigan, United States
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment
Change from Baseline to Week 27 in the area under the curve (AUC) of Plasma Glucose was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of Serum Insulin was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of Serum C-peptide was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of FFA was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of Serum Ghrelin was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
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Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of Incretin Hormone: GIP was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of Incretin Hormone: GLP-1 was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment
Change from Baseline to Week 27 in the AUC of PYY was assessed.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in HDL-C at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in LDL-C at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Total Cholesterol (TC) at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in VLDL-C at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Non-HDL-C at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoB at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoB-48 at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoA-1 at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: Chylomicron at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: VLDL at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: LDL at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: HDL at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Glycerol Levels at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
The baseline was defined as the Day 1 pre-dose fasting assessment. Lipoprotein Particle size included: HDL size, LDL size and VLDL size.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment
The baseline was defined as the last non-missing assessment prior to the first dose of study drug.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR)
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Adiponectin at End of the Treatment
The baseline was defined as the Day 1 pre-dose fasting assessment.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in and Leptin at End of the Treatment
The baseline was defined as the Day 1 pre-dose fasting assessment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment
The baseline was defined as the last non-missing assessment prior to the first dose of study drug.
Time frame: Baseline and End of the Treatment (Week 27)
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 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
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.
Time frame: Baseline and End of the Treatment (Week 27)
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
The baseline was defined as Screening assessment. Change in body fat distribution for total Bone mineral density was measures obtained from DEXA.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Visceral Adipose Tissue (VAT) as Measured by Magnetic Resonance Imaging (MRI) at End of the Treatment
The baseline was defined as the last assessment prior to the first dose of study drug.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Subcutaneous Adipose Tissue (SAT) as MRI at End of the Treatment
The baseline was defined as the last assessment prior to the first dose of study drug.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Body Weight at End of the Treatment
The baseline was defined as the Day 1 pre-dose assessment.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Waist Circumference at End of the Treatment
The baseline was defined as the Screening assessment.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Waist/Hip Ratio at End of the Treatment
The baseline was defined as screening assessment.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Quality of Life (QoL)
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.
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Pain Score at End of the Treatment
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.
Time frame: Baseline and End of the Treatment (Week 27)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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
Time frame: From signing of informed consent to end of follow up period (Up to week 40)