Back to resultsStudy of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Primary Purpose
Familial Partial Lipodystrophy
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
AKCEA-ANGPTL3-LRx
Sponsored by
About this trial
This is an interventional treatment trial for Familial Partial Lipodystrophy focused on measuring Lipodystrophy, Lipodystrophy, Familial Partial, Lipid Metabolism Disorders, Dyslipidemias, Kobberling-Dunnigan syndrome (type 1 and 2), Lipoatrophic Diabetes
Eligibility Criteria
Key Inclusion Criteria:
- Must give written informed consent to participate in the study.
- Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
- Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol.
- Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams per deciliter (mg/dL) at both Screening and Qualification visits. Participants with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per liter [mmol/L]) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history criteria for study inclusion may be further screened and enrolled in the study.
- Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.
Key Exclusion Criteria:
- Diagnosis of generalized lipodystrophy.
- Diagnosis of acquired partial lipodystrophy (APL).
- Acute pancreatitis within 4 weeks of Screening.
- Acute coronary syndrome within 6 months of Screening.
- Major surgery within 3 months of Screening.
- Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.
Sites / Locations
- Clinical Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AKCEA-ANGPTL3-LRx 20 mg
Arm Description
Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Change From Baseline in Adiponectin at End of the Treatment
The baseline was defined as the Day 1 pre-dose fasting assessment.
Change From Baseline in and Leptin at End of the Treatment
The baseline was defined as the Day 1 pre-dose fasting assessment
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.
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.
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.
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.
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.
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.
Change From Baseline in Body Weight at End of the Treatment
The baseline was defined as the Day 1 pre-dose assessment.
Change From Baseline in Waist Circumference at End of the Treatment
The baseline was defined as the Screening assessment.
Change From Baseline in Waist/Hip Ratio at End of the Treatment
The baseline was defined as screening assessment.
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.
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.
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
Full Information
NCT ID
NCT03514420
First Posted
April 21, 2018
Last Updated
January 27, 2021
Sponsor
Akcea Therapeutics
Collaborators
Ionis Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03514420
Brief Title
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Official Title
An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
June 15, 2018 (Actual)
Primary Completion Date
July 30, 2019 (Actual)
Study Completion Date
August 21, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Akcea Therapeutics
Collaborators
Ionis Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Partial Lipodystrophy
Keywords
Lipodystrophy, Lipodystrophy, Familial Partial, Lipid Metabolism Disorders, Dyslipidemias, Kobberling-Dunnigan syndrome (type 1 and 2), Lipoatrophic Diabetes
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AKCEA-ANGPTL3-LRx 20 mg
Arm Type
Experimental
Arm Description
Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection.
Intervention Type
Drug
Intervention Name(s)
AKCEA-ANGPTL3-LRx
Other Intervention Name(s)
ISIS 703802
Intervention Description
AKCEA-ANGPTL3-LRx solution for SC injection.
Primary Outcome Measure Information:
Title
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
Description
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)
Secondary Outcome Measure Information:
Title
Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment
Description
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)
Title
Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment
Description
Change from Baseline to Week 27 in the AUC of Serum Insulin was assessed.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment
Description
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)
Title
Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment
Description
Change from Baseline to Week 27 in the AUC of FFA was assessed.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment
Description
Change from Baseline to Week 27 in the AUC of Serum Ghrelin was assessed.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment
Description
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)
Title
Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment
Description
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)
Title
Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment
Description
Change from Baseline to Week 27 in the AUC of PYY was assessed.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in HDL-C at End of the Treatment
Description
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)
Title
Change From Baseline in LDL-C at End of the Treatment
Description
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)
Title
Change From Baseline in Total Cholesterol (TC) at End of the Treatment
Description
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)
Title
Change From Baseline in VLDL-C at End of the Treatment
Description
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)
Title
Change From Baseline in Non-HDL-C at End of the Treatment
Description
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)
Title
Change From Baseline in ApoB at End of the Treatment
Description
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)
Title
Change From Baseline in ApoB-48 at End of the Treatment
Description
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)
Title
Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment
Description
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)
Title
Change From Baseline in ApoA-1 at End of the Treatment
Description
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)
Title
Change From Baseline in ApoC-III at End of the Treatment
Description
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)
Title
Change From Baseline in ApoC-III: Chylomicron at End of the Treatment
Description
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)
Title
Change From Baseline in ApoC-III: VLDL at End of the Treatment
Description
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)
Title
Change From Baseline in ApoC-III: LDL at End of the Treatment
Description
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)
Title
Change From Baseline in ApoC-III: HDL at End of the Treatment
Description
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)
Title
Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment
Description
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)
Title
Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment
Description
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)
Title
Change From Baseline in Glycerol Levels at End of the Treatment
Description
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)
Title
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
Description
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)
Title
Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment
Description
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)
Title
Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR)
Description
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)
Title
Change From Baseline in Adiponectin at End of the Treatment
Description
The baseline was defined as the Day 1 pre-dose fasting assessment.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in and Leptin at End of the Treatment
Description
The baseline was defined as the Day 1 pre-dose fasting assessment
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment
Description
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)
Title
Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment
Description
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)
Title
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
Description
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)
Title
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
Description
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)
Title
Change From Baseline in Visceral Adipose Tissue (VAT) as Measured by Magnetic Resonance Imaging (MRI) at End of the Treatment
Description
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)
Title
Change From Baseline in Subcutaneous Adipose Tissue (SAT) as MRI at End of the Treatment
Description
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)
Title
Change From Baseline in Body Weight at End of the Treatment
Description
The baseline was defined as the Day 1 pre-dose assessment.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in Waist Circumference at End of the Treatment
Description
The baseline was defined as the Screening assessment.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in Waist/Hip Ratio at End of the Treatment
Description
The baseline was defined as screening assessment.
Time Frame
Baseline and End of the Treatment (Week 27)
Title
Change From Baseline in Quality of Life (QoL)
Description
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)
Title
Change From Baseline in Pain Score at End of the Treatment
Description
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)
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
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)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Must give written informed consent to participate in the study.
Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol.
Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams per deciliter (mg/dL) at both Screening and Qualification visits. Participants with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per liter [mmol/L]) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history criteria for study inclusion may be further screened and enrolled in the study.
Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.
Key Exclusion Criteria:
Diagnosis of generalized lipodystrophy.
Diagnosis of acquired partial lipodystrophy (APL).
Acute pancreatitis within 4 weeks of Screening.
Acute coronary syndrome within 6 months of Screening.
Major surgery within 3 months of Screening.
Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.
Facility Information:
Facility Name
Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
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