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

Overview

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

A.J. Vinaya Simha, M.B.B.S., M.D.

Closed for enrollment

More information

Publications

Publications are currently not available

Additional contact information

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Phone: 800-664-4542 (toll-free)

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