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The BROADEN Study: A Study of Volanesorsen (Formerly ISIS-APOCIIIRx) in Patients With Familial Partial Lipodystrophy

This study is currently recruiting participants.
Verified November 2017 by Ionis Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02527343
First Posted: August 19, 2015
Last Update Posted: November 14, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
  Purpose
The purpose of this study is to evaluate the efficacy and safety of volanesorsen (IONIS-APOCIIIRx) given for 52 weeks in patients with Familial Partial Lipodystrophy. Patients will then be allowed to continue in a 2 year Open Label Extension of the study.

Condition Intervention Phase
Familial Partial Lipodystrophy Drug: volanesorsen Drug: Placebo Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, With an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Partial Lipodystrophy

Resource links provided by NLM:


Further study details as provided by Ionis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Efficacy of volanesorsen (IONIS-APOCIIIRx) measured as the percent change in fasting triglycerides from baseline. [ Time Frame: 13 weeks ]

Secondary Outcome Measures:
  • Change from Baseline in hepatic steatosis (as assessed by hepatic fat fraction using magnetic resonance imaging (MRI). [ Time Frame: 52 weeks ]
  • Change from Baseline in hemoglobin A1c (HbA1c) [ Time Frame: 52 weeks ]
  • A composite endpoint at Month 6 for percent of patients who achieve a. ≥ 40% reduction in fasting TG, and b. ≥ 30% reduction of hepatic fat fraction percent [ Time Frame: 52 weeks ]
  • Change in patient-reported outcomes (PRO) [ Time Frame: 52 weeks ]

Estimated Enrollment: 60
Actual Study Start Date: October 31, 2015
Estimated Study Completion Date: September 30, 2021
Estimated Primary Completion Date: September 30, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: volanesorsen 300mg
volanesorsen administered subcutaneously once weekly for 52 weeks
Drug: volanesorsen
Other Name: ISIS 304801, IONIS-APOCIIIRx
Placebo Comparator: Placebo
Placebo administered subcutaneously once weekly for 52 weeks.
Drug: Placebo
Active Comparator: Open Label Extension (OLE)
Volanesorsen administered subcutaneously once weekly for 104 weeks
Drug: volanesorsen
Other Name: ISIS 304801, IONIS-APOCIIIRx

  Eligibility

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Must give written informed consent to participate in the study (signed and dated) and any authorizations required by law
  • Clinical diagnosis of FPL plus diagnosis of type 2 diabetes mellitus, hypertriglyceridemia, and fatty liver

    • Diagnosis of FPL is based on deficiency of subcutaneous body fat in a partial fashion assessed by physical examination and low skinfold thickness in anterior thigh by caliper measurement: men (≤ 10 mm) and women (≤ 22 mm), and at least 1 of the following:

      1. Genetic diagnosis of FPL OR
      2. Family history of FPL or of similar abnormal fat distribution plus 1 Minor Criteria OR
      3. In the absence of FPL-associated genetic variant or family history, 2 Minor Criteria and BMI< 35 kg/m2
    • Diabetes not well controlled on antidiabetic therapy with HbA1c ≥ 7% to ≤ 12% at Screening
    • Hypertriglyceridemia with Fasting TG levels ≥ 500 mg/dL (≥ 5.7 mmol/L) at Screening and Qualification visit, or Fasting TG levels ≥ 200 (≥ 2.26 mmol/L) at both Screening and Qualification Visits for patients who meet the genetic or family history criteria
    • Presence of hepatosteatosis (fatty liver), as evidenced by a Screening MRI indicating a hepatic fat fraction (HFF) ≥ 6.4%.

Exclusion Criteria:

  • A diagnosis of generalized lipodystrophy
  • A diagnosis of acquired partial lipodystrophy
  • Acute pancreatitis within 4 weeks of Screening
  • History within 6 months of Screening of acute or unstable cardiac condition
  • LDL-C > 130 mg/dL on maximal tolerated statin therapy
  • Platelet count < lower limit of normal (LLN)
  • Treatment with metreleptin within the last 3 months prior to Screening
  Contacts and Locations
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): NCT02527343


Contacts
Contact: Patient Advocacy 442-222-4685 patientadvocacy@akceatx.com

  Show 36 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
  More Information

Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02527343     History of Changes
Other Study ID Numbers: ISIS 304801-CS17
First Submitted: August 17, 2015
First Posted: August 19, 2015
Last Update Posted: November 14, 2017
Last Verified: November 2017

Additional relevant MeSH terms:
Lipodystrophy
Lipodystrophy, Familial Partial
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases