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Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03514420
Recruitment Status : Completed
First Posted : May 2, 2018
Last Update Posted : September 13, 2019
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Akcea Therapeutics

Tracking Information
First Submitted Date  ICMJE April 21, 2018
First Posted Date  ICMJE May 2, 2018
Last Update Posted Date September 13, 2019
Actual Study Start Date  ICMJE May 30, 2018
Actual Primary Completion Date July 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 21, 2018)
Reduction of fasting triglycerides (TG). [ Time Frame: 27 weeks ]
Percent change in fasting TG from from Baseline to Week 27.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03514420 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 21, 2018)
  • Effect of ISIS 703802 on mixed meal test (MMT). [ Time Frame: 27 weeks ]
    Change in glucose parameters in response to a mixed meal test (MMT) from Baseline to Week 27 will be summarized.
  • Effect of ISIS 703802 on lipid parameters. [ Time Frame: 27 weeks ]
    Absolute and percentage change on lipid parameters from Baseline to Week 27 will be summarized.
  • Effect of ISIS 703802 on glycosylated hemoglobin (HbA1c). [ Time Frame: 27 weeks ]
    Change from Baseline in HbA1c at Week 27.
  • Effect of ISIS 703802 on hepatic fat fraction. [ Time Frame: 27 weeks ]
    Change from Baseline in hepatic fat fraction (as assessed by magnetic resonance imaging [MRI]) at Week 27.
  • Effect of ISIS 703802 on fat distribution in various areas of the body. [ Time Frame: 27 weeks ]
    Changes Changes from Baseline in body fat distribution for various areas in the body as measured by skinfold thickness and DEXA at Week 27.
  • The safety of ISIS 703802 by the incidence of treatment-emergent adverse events. [ Time Frame: 27 weeks ]
    The safety of ISIS 703802 will be assessed by determining adverse effects.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)
Official Title  ICMJE An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
Brief Summary This is a multi-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of fasting triglycerides in patients with Familial Partial Lipodystrophy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Familial Partial Lipodystrophy
Intervention  ICMJE Drug: AKCEA-ANGPTL3-LRX
Single open-label cohort
Other Name: ISIS 703802
Study Arms  ICMJE Experimental: AKCEA-ANGPTL3-LRX Dose 1
Intervention: Drug: AKCEA-ANGPTL3-LRX
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 11, 2019)
4
Original Estimated Enrollment  ICMJE
 (submitted: April 21, 2018)
3
Actual Study Completion Date  ICMJE August 20, 2019
Actual Primary Completion Date July 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 HbA1c ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol
  • Hypertriglyceridemia as defined by fasting TG levels ≥ 500 mg/dL at both Screening and Qualification visits. Patients with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 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 MRI indicating a hepatic fat fraction (HFF) ≥ 6.4%

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 patient participating in or completing the study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03514420
Other Study ID Numbers  ICMJE ISIS 703802-CS5
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Akcea Therapeutics
Study Sponsor  ICMJE Akcea Therapeutics
Collaborators  ICMJE Ionis Pharmaceuticals, Inc.
Investigators  ICMJE Not Provided
PRS Account Akcea Therapeutics
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP