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

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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

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.

Condition or disease Intervention/treatment Phase
Familial Partial Lipodystrophy Drug: AKCEA-ANGPTL3-LRX Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
Actual Study Start Date : May 30, 2018
Actual Primary Completion Date : July 30, 2019
Actual Study Completion Date : August 20, 2019


Arm Intervention/treatment
Experimental: AKCEA-ANGPTL3-LRX Dose 1 Drug: AKCEA-ANGPTL3-LRX
Single open-label cohort
Other Name: ISIS 703802




Primary Outcome Measures :
  1. Reduction of fasting triglycerides (TG). [ Time Frame: 27 weeks ]
    Percent change in fasting TG from from Baseline to Week 27.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. Effect of ISIS 703802 on glycosylated hemoglobin (HbA1c). [ Time Frame: 27 weeks ]
    Change from Baseline in HbA1c at Week 27.

  4. 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.

  5. 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.

  6. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03514420


Locations
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United States, Michigan
Clinical Site
Ann Arbor, Michigan, United States, 48105
Sponsors and Collaborators
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

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Responsible Party: Akcea Therapeutics
ClinicalTrials.gov Identifier: NCT03514420     History of Changes
Other Study ID Numbers: ISIS 703802-CS5
First Posted: May 2, 2018    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Akcea Therapeutics:
Lipodystrophy
Lipodystrophy, Familial Partial
Lipid Metabolism Disorders
Dyslipidemias
Kobberling-Dunnigan syndrome (type 1 and 2)
Lipoatrophic Diabetes
Additional relevant MeSH terms:
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Lipodystrophy
Lipodystrophy, Familial Partial
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases