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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.
See Contacts and Locations
Verified July 2017 by Ionis Pharmaceuticals, Inc.
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02527343
First received: August 17, 2015
Last updated: July 10, 2017
Last verified: July 2017
  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.

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 hemoglobin A1c (HbA1c) in patients with diabetes. [ Time Frame: 13 weeks ]

Estimated Enrollment: 60
Actual Study Start Date: October 31, 2015
Estimated Primary Completion Date: September 30, 2017 (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 52 weeks
Drug: volanesorsen
Other Name: ISIS 304801, IONIS-APOCIIIRx

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of FPL plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
  • Diagnosis of FLP 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:
  • Genetic diagnosis of familial PL (e.g., mutations in LMNA, PPAR-γ, AKT2, CIDEC, PLIN1 genes)

OR

-Family history of FPL or family history of abnormal and similar fat distribution plus 1 Minor Criteria

OR

-2 Minor Criteria (In the absence of FPL-associated genetic variant or family history) and BMI< 35 kg/m2

Minor Criteria

  1. Requirement for high doses of insulin, e.g., requiring ≥ 200 U/day, ≥ 2 U/kg/day, or currently taking U-500 insulin
  2. Presence of acanthosis nigricans on physical examination
  3. Evidence/history of polycystic ovary syndrome (PCOS) or PCOS-like symptoms (hirsutism, oligomenorrhea, and/or polycystic ovaries)
  4. History of pancreatitis associated with hypertriglyceridemia
  5. Evidence of non-alcoholic fatty liver disease

    • Hepatomegaly and/or elevated transaminases in the absence of a known cause of liver disease or radiographic evidence of hepatic steatosis (e.g., on ultrasound or CT)
    • Diagnosis of Type 2 diabetes mellitus as defined by the American Diabetes Association Standards of Medical Care in Diabetes 2015) made at least 12 weeks prior to the Screening visit, and 2 of the following:

      • on standard-of-care anti-diabetic therapy, and
      • HbA1c ≥ 7% to ≤ 10% at Screening.
    • Hypertriglyceridemia is defined as Fasting TG levels ≥ 500 mg/dL (≥ 5.7 mmol/L) at Screening and Qualification visit.

      • If the fasting TG value at Screening and/or Qualification visit is < 500 mg/dL (< 5.7 mmol/L) but ≥ 350 mg/dL (≥ 4.0 mmol/L) up to 2 additional tests may be performed in order to qualify

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 ischemia (myocardial infarction, acute coronary syndrome, new onset angina), stroke, transient ischemic attack, or unstable congestive heart failure requiring a change in medication
    • Major surgery within 3 months of Screening
    • Treatment with Metreleptin within the last 3 months prior to Screening
    • Previous treatment with ISIS-APOCIIIRx
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02527343

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

  Show 34 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
Study First Received: August 17, 2015
Last Updated: July 10, 2017

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

ClinicalTrials.gov processed this record on September 19, 2017