Familial Partial LipodystrophyPhase 2NCT03514420
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
A Phase 2 study testing AKCEA-ANGPTL3-LRx in people with Familial Partial Lipodystrophy, run by Akcea Therapeutics at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Partial Lipodystrophy
- What is being tested?
- AKCEA-ANGPTL3-LRx
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2018to 21 Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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%.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
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.
- Conditions
- Familial Partial Lipodystrophy
- Being tested
- AKCEA-ANGPTL3-LRx
- Keywords
- Lipodystrophy, Lipodystrophy, Familial Partial, Lipid Metabolism Disorders, Dyslipidemias, Kobberling-Dunnigan syndrome (type 1 and 2), Lipoatrophic Diabetes
- Medical subject headings
- Lipodystrophy, Familial Partial, Lipodystrophy, Lipid Metabolism Disorders, Dyslipidemias, Diabetes Mellitus, Lipoatrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AKCEA-ANGPTL3-LRxDrug
Also called ISIS 703802
AKCEA-ANGPTL3-LRx solution for SC injection.
| Study group | Role | What they receive |
|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection. Receives: Drug: AKCEA-ANGPTL3-LRx |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and End of the Treatment (Week 27)
Secondary outcomes (42)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline and End of the Treatment (Week 27)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: From signing of informed consent to end of follow up period (Up to week 40)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2018
- Study start15 Jun 2018
- Primary completion30 Jul 2019
- Study completion21 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Familial Partial LipodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03514420
- Sponsor's protocol ID
- ISIS 703802-CS5
- Status last verified
- Jan 2021
- First submitted
- 21 Apr 2018
- Last updated
- 16 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
- ClinicalTrials.gov ID
- NCT03514420
- Acronym / sponsor ID
- ISIS 703802-CS5
- Conditions
- Familial Partial Lipodystrophy
- Keywords
- Lipodystrophy, Lipodystrophy, Familial Partial, Lipid Metabolism Disorders, Dyslipidemias, Kobberling-Dunnigan syndrome (type 1 and 2), Lipoatrophic Diabetes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Study start
- 15 Jun 2018 (actual)
- Primary completion
- 30 Jul 2019 (actual)
- Study completion
- 21 Aug 2019 (actual)
- First posted
- 2 May 2018 (actual)
- Results
- Posted 16 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Drug: AKCEA-ANGPTL3-LRxParticipants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AKCEA-ANGPTL3-LRxAlso: ISIS 703802AKCEA-ANGPTL3-LRx solution for SC injection. | Drug | AKCEA-ANGPTL3-LRx 20 mg |
Primary outcome
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
Time frame: Baseline and 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.
42 secondary outcomes
Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment
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
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
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
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
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment
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
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
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in HDL-C at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in LDL-C at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Total Cholesterol (TC) at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in VLDL-C at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Non-HDL-C at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoB at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoB-48 at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoA-1 at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: Chylomicron at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: VLDL at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: LDL at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in ApoC-III: HDL at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Glycerol Levels at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Lipoprotein Particle Size at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR)
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Adiponectin at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in and Leptin at End of the Treatment
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
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
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
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
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
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
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Body Weight at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Waist Circumference at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Waist/Hip Ratio at End of the Treatment
Time frame: Baseline and End of the Treatment (Week 27)
Change From Baseline in Quality of Life (QoL)
Time frame: Baseline and End of the Treatment (Week 27)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AKCEA-ANGPTL3-LRx in people with Familial Partial Lipodystrophy, run by Akcea Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27) (time frame: Baseline and End of the Treatment (Week 27)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Feb 2021.